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The FDA approved olaparib (Lynparza; AstraZeneca) for the treatment of patients with advanced ovarian cancer who have a deleterious or suspected deleterious germline BRCA mutation and who have been treated with three or more lines of chemotherapy. Concurrently, the FDA approved the BRACAnalysis test (Myriad Genetics) as a companion diagnostic to identify patients with BRCA1 and BRCA2 germline mutations. Olaparib is a poly ADP-ribose polymerase (PARP) inhibitor that blocks enzymes involved in repairing damaged DNA.
Osaka, Japan's ONO Pharmaceutical and Foster City, CA's Gilead Sciences announced that they will jointly develop and commercialize ONO-4059, ONO's investigational Bruton's tyrosine kinase inhibitor for the treatment of B-cell malignancies and other diseases. Gilead said it will pay ONO an upfront fee and make additional payments based upon achievement of certain milestones but did not disclose the amount of those payments.
Reuters reported that Swiss drugmaker Roche will make payments totaling up to $489 million to acquire Dutalys, an Austrian biotech company that develops bispecific antibodies. Bispecific antibodies are composed of fragments of two different monoclonal antibodies, allowing them to bind to two different antigens.
Although very small, the risk of developing leukemia after radiotherapy or chemotherapy for early-stage breast cancer is twice as high as previously reported, according to results of a study led by researchers at the Johns Hopkins Kimmel Cancer Center in Baltimore, MD, and reported in the Journal of Clinical Oncology. Earlier, smaller trials found that about 0.25% of breast cancer patients develop leukemia as a late effect of treatment, but the new analysis of 20,063 patients puts the risk at about 0.5%.
In addition, the FDA approved nivolumab (Opdivo; Bristol-Myers Squibb) for the treatment of patients with inoperable or metastatic melanoma who no longer respond to other drugs, and whose tumors express the BRAF V600 gene mutation. Nivolumab inhibits the PD-1 protein on cells, which blocks the body's immune system from attacking melanoma cells.
The European Commission granted marketing authorization to ramucirumab (Cyramza) for the treatment of advanced stomach and gastroesophageal cancers. A VEGF inhibitor, Eli Lilly's ramucirumab is approved in the United States for these cancers, as well as for non–small cell lung cancer. Based on studies showing improved overall survival rates, ramucirumab may soon garner FDA approval for use in metastatic colorectal cancer.
An analysis of 29,254 women published in Cancer suggests that women who use bisphosphonates have about half the risk of developing endometrial cancer as women who do not use these drugs. Bisphosphonates are commonly prescribed to treat osteoporosis and other bone conditions. These findings support other research that has shown that the drugs have an anticancer effect.
BGI-Shenzhen and South University of Science and Technology of China, both in Shenzhen, China, and Denmark's University of Copenhagen agreed to establish a specialized college in Shenzhen. According to BGI-Shenzhen, the college will "focus on cultivating world-class, high-end talents for the life sciences and bio-industry, producing scientific research achievements with international influence and boosting the development of the bio-industry in China."
The FDA approved Ipsen Pharma's lanreotide (Somatuline depot injection) to treat unresectable, well or moderately differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors and improve progression-free survival. Lanreotide was previously approved for the long-term treatment of patients with acromegaly, a condition in which the pituitary gland produces too much growth hormone, that doesn't respond to surgery or radiotherapy.
The FDA also approved ramucirumab (Cyramza; Eli Lilly) to treat patients with metastatic non–small cell lung cancer whose tumor has progressed during or after treatment with platinum-based chemotherapy. The drug, which blocks the blood supply that fuels tumor growth, was approved earlier this year, both alone and in combination with paclitaxel, to treat patients with advanced stomach cancer or gastroesophageal junction adenocarcinoma.
The U.S. Court of Appeals for the Federal Circuit upheld an earlier U.S. District Court decision denying Myriad Genetics' request for an injunction against Ambry Genetics to prevent it from offering gene tests for BRCA1 and BRCA2. Salt Lake City, UT–based Myriad sued Ambry (Aliso Viejo, CA) in July 2013 alleging patent infringement on the gene tests, which Ambry began offering after the U.S. Supreme Court ruled a month earlier that human genes cannot be patented; synthetic DNA, which does not occur naturally, can be patented.
By a vote of 51–43, the U.S. Senate confirmed Vivek Murthy, MD, MBA, as surgeon general. Murthy is a faculty member at Harvard Medical School and a hospitalist at Brigham and Women's Hospital in Boston, MA. Boris Lushniak, MD, MPH, has been serving as acting surgeon general.
The European Commission granted marketing authorization for olaparib (Lynparza; AstraZeneca) for maintenance treatment of patients with platinum-sensitive, relapsed BRCA-mutated, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancers who experienced a complete or partial response to platinum-based chemotherapy. A PARP inhibitor, olaparib exploits tumor DNA repair pathway deficiencies to preferentially kill cancer cells.
In an updated policy statement published in the Journal of Clinical Oncology, the American Society of Clinical Oncology called for greater access to and education about phase I clinical trials. Barriers to clinical trial participation, such as the lack of insurance coverage for routine care in clinical trials and the need to improve clinicians' ability to talk with patients about the risks and benefits of trials, should be addressed, according to the statement.
In response to an advisory committee's report, the NIH announced the cancellation of the National Children's Study (NCS), which had been designed to examine the effects of environmental and biologic factors on children's health and development by following 100,000 individuals from birth to age 21. Although the committee praised the NCS's goals, it noted concerns about the study's design and increasing costs; despite initiating a pilot effort and spending more than $1 billion over several years, the NCS never started. "I am disappointed that this study failed to achieve its goals," said NIH Director Francis Collins, MD, PhD. "Yet I am optimistic that other approaches will provide answers to these important research questions."
The FDA approved Gardasil 9 (Merck Sharp & Dohme), a vaccine for the prevention of certain diseases caused by nine types of human papillomavirus (HPV), five more than the previously approved Gardasil. Approved for use in females ages 9 through 26 and males ages 9 through 15, Gardasil 9 has the potential to prevent approximately 90% of cervical, vulvar, vaginal, and anal cancers, including those caused by HPV types 16, 18, 31, 33, 45, 52, and 58, and to prevent genital warts caused by HPV types 6 or 11.
The U.S. Congress reached a deal on a $1.013 trillion budget package that will fund the federal government through September 2015. The budget includes an increase of just $150 million (0.5%) for NIH over fiscal year 2014, to $30.084 billion, and an increase of just $27 million (0.54%) for NCI, to $4.950 billion, amounts that do not keep pace with inflation. "We appreciate any increase, but it's not getting the job done," Jennifer Zeitzer, deputy director of the Federation of American Societies for Experimental Biology's Office of Public Affairs, told ScienceInsider. "We're going backwards," she said.
A Russian billionaire bought James Watson's Nobel Prize at auction for $4.1 million, even more than presale estimates suggesting that the medallion could fetch $3.5 million. Various media reports say that the buyer will return the medal to Watson, who shared the 1962 award for his contributions to the discovery of the structure of DNA.
A study of 7,154 pre- and postmenopausal women at high risk of breast cancer found that women who took tamoxifen for 5 years reduced their risk of developing the disease by about 30% over 20 years, according to data presented at the San Antonio Breast Cancer Symposium in Texas on December 11 and published concurrently in The Lancet Oncology. "We hope these results will stimulate more women, particularly younger women, to consider treatment options for breast cancer prevention if they have a family history of the disease or other major risk factors," said Jack Cuzick, PhD, professor of epidemiology at Queen Mary University of London, UK, who presented the findings.
In a phase I study of the IDH2 inhibitor AG-221 (Agios Pharmaceuticals) in 45 patients with relapsed or unresponsive IDH2-positive advanced hematologic malignancies, 33% of patients achieved a complete remission, 22% achieved partial remission, and 38% had stable disease. "We haven't yet reached the maximum tolerated dose and patients are responding dramatically," said Eytan Stein, MD, a medical oncologist at Memorial Sloan Kettering Cancer Center in New York, NY, who presented the findings at the annual meeting of the American Society of Hematology in San Francisco, CA. "More research is needed, but I am optimistic that this drug will fundamentally alter the natural history of IDH2-mutant leukemia and other hematologic malignancies."
The FDA granted accelerated approval to blinatumomab (Blincyto; Amgen) to treat patients with Philadelphia chromosome–negative relapsed or refractory precursor B-cell acute lymphoblastic leukemia (B-cell ALL), an uncommon form of ALL. Blinatumomab is a bispecific CD19-directed CD3 T-cell engager that activates endogenous T cells when bound to the CD19-expressing target cell. Safety and effectiveness of the drug were evaluated in a clinical study involving 185 adults with B-cell ALL, 32% of whom experienced complete remission for a median of 6.7 months.
The NCI issued its "Annual Plan and Budget Proposal for Fiscal Year 2016," which describes current opportunities to advance cancer research and calls for strong and sustained funding. The "professional judgment" budget recommendation includes increases in funding that will "enable the NCI to address today's most promising opportunities in cancer research." The Institute calls for $5.75 billion in funding for fiscal year 2016, an amount $1 billion below what it would have been if budgets had kept pace with inflation over the past decade.
The World Health Organization (WHO) issued a new guidance document to help countries better prevent and control cervical cancer. "Comprehensive Cervical Cancer Control: A guide to essential practice" calls for vaccinating girls ages 9 to 13 with two doses of the human papillomavirus (HPV) vaccine, using HPV tests to screen women for cervical cancer, and communicating more widely about the importance of cervical cancer screening.
Puma Biotechnology announced that it will delay filing for FDA approval of neratinib for HER2-positive metastatic breast cancer until early 2016 instead of in the first half of 2015. The company said it will first apply for approval to use the drug in HER2-positive early-stage disease instead. Puma is also testing neratinib for use in other types of breast cancer, lung cancer, and other malignancies.
Nearly half a million cancer cases a year worldwide can be attributed to overweight and obesity, according to a study by the International Agency for Research on Cancer, an agency of the WHO. Published in The Lancet Oncology, the study estimates that these risk factors were responsible for about 3.6% of all new cancer cases in 2012. North America continues to be most affected, with an estimated 111,000 obesity-related cancer deaths in 2012, accounting for 23% of the global total.
The NIH issued a draft policy to promote the use of single institutional review boards (IRB) in multisite clinical research studies. Multisite studies can often generate results in less time. However, working through IRB review at each site can add delay without increasing the protections for the research participants. Exceptions to the policy would be allowed if local IRB review is necessary to meet the needs of specific populations or where it is required by federal, state, or tribal laws or regulations.
Astellas Pharma Europe announced that enzalutamide (Xtandi) has been approved in Europe for the treatment of men with metastatic castration-resistant prostate cancer after androgen deprivation therapy has failed and in whom chemotherapy is not yet clinically indicated. In a phase III trial, enzalutamide, which inhibits androgen-receptor signaling, reduced the risk of death by 29% and the risk of radiographic progression or death by 81% compared with a placebo. Men who took the drug also delayed the start of chemotherapy by 17 months compared with those taking a placebo.
Amgen announced the termination of its clinical studies of rilotumumab in advanced gastric cancer. The decision was based on a safety review by an independent data-monitoring committee that found an increase in the number of deaths in the rilotumumab-plus-chemotherapy arm when compared with the chemotherapy-only arm. The company noted that futility criteria would likely have been met at the planned interim analysis in March 2015.
The FDA recommended that makers of laparoscopic power morcellators include a boxed warning that the use of the devices may spread unsuspected cancer in women being treated for uterine fibroids and decrease their long-term survival. The agency also said that product labeling should note that the devices are contraindicated for the removal of fibroids in peri- and postmenopausal women and in women who undergo hysterectomy or myomectomy, as well as in cases where the tissue is suspected to be cancerous. Younger women who want to keep their uterus intact may opt for the procedure after being fully informed of the risks.
Early next month, Christie's auction house in New York, NY, will sell James Watson's Nobel Prize at auction, along with some of his notes for his acceptance speech and a manuscript of his Nobel address, according to Reuters. Proceeds from the sale, which could total more than $3.5 million, will be donated to charity and scientific research.
Maryland's Baltimore Business Journal reported that "AstraZeneca will expand its biologics manufacturing center in Frederick and add hundreds of jobs to its operations there." The company is expected to "spend more than $200 million to increase production capacity." The expansion is slated to begin next month and last more than 2 years.
Cambridge, MA–based Merrimack Pharmaceuticals announced that the FDA granted Fast Track designation to MM-398 for the treatment of patients with metastatic adenocarcinoma of the pancreas who have been previously treated with gemcitabine-based therapy. MM-398 is a nanoliposomal encapsulation of the chemotherapeutic irinotecan. The designation expedites the development and review of drugs that treat serious conditions and fill an unmet medical need.
To help ensure transparency around NIH-supported research, the agency proposed expanding the scope of clinical trials required to submit summary results to include trials of unapproved, unlicensed, and uncleared products. "If the clinical research community fails to share what is learned," wrote NIH director Francis Collins, MD, PhD, and Kathy Hudson, PhD, in the Journal of the American Medical Association, "… researchers are reneging on the promise to clinical trial participants, are wasting time and resources, and are jeopardizing public trust." A summary of the proposed changes is available at www.nih.gov/.
The American Society of Clinical Oncology announced plans to launch a study of molecularly targeted cancer drugs used off-label for the treatment of patients with advanced solid tumors with a known genomic variant. Called the Targeted Agent and Profiling Utilization Registry (TAPUR) study, the project will be the first clinical trial ever led by the society. The goal of TAPUR is to describe the antitumor activity and toxicity of the therapies and to help patients who no longer benefit from standard treatment to obtain commercially available therapies for off-label use.
Pfizer and Merck KGaA signed a deal worth up to $2.85 billion to collaborate on the development and marketing of immunotherapies for cancer. Merck KGaA will receive $850 million upfront and up to $2 billion in milestone payments for the anti–PD-L1 therapy MSB0010718C and any other immuno-oncology drugs it adds to its pipeline. The companies will also work together to bring Pfizer's preclinical anti–PD-1 antibody into phase I trials and promote Pfizer's ALK inhibitor crizotinib (Xalkori).
The FDA approved bevacizumab (Avastin) in combination with chemotherapy for the treatment of platinum-resistant ovarian, fallopian tube, and primary peritoneal cancers, Genentech announced. The approval was based on results from the phase III AURELIA study that showed bevacizumab plus chemotherapy reduced the risk of disease worsening or death by 62 percent compared with chemotherapy alone. The median progression-free survival (PFS) was 6.8 months for the combination and 3.4 months for chemotherapy alone.
In a phase II trial of neratinib plus paclitaxel versus trastuzumab (Herceptin; Genentech/Roche) plus paclitaxel in patients with HER2+ breast cancer, neratinib and trastuzumab yielded similar PFS (16.6 months vs. 16.7 months) and objective response rates (ORR; 74.8% vs. 75.1%), according to Puma Biotechnology, neratinib's manufacturer. However, the incidence of brain metastases was 52.6% lower among patients who received neratinib compared with those who received trastuzumab (7.4% vs. 15.6%).
Seattle, WA–based Juno Therapeutics announced that the FDA granted Orphan Drug designation to JCAR015, a chimeric antigen receptor T-cell product, for the treatment of acute lymphoblastic leukemia. The company also filed paperwork for a $150 million initial public offering of stock. It plans to start trading on NASDAQ under JUNO.
Threshold Pharmaceuticals announced that the FDA granted Fast Track designation to TH-302 for the treatment of certain patients with soft tissue sarcoma. The South San Francisco, CA, company said that the drug is being evaluated in a phase III trial in combination with doxorubicin, compared with doxorubicin alone. The primary endpoint of the trial is overall survival; secondary endpoints include PFS and ORR.
Los Angeles, CA–based CytRx said that the FDA has placed a partial clinical hold on its trials of aldoxorubicin, which is under investigation for the treatment of Kaposi sarcoma, glioblastoma multiforme, and soft tissue sarcoma. Patients currently enrolled in a clinical trial can continue receiving the drug, or comparator drugs, but no new patients can be enrolled until the clinical hold is lifted. According to CytRx, the hold is due to the death of a patient who received aldoxorubicin under the company's compassionate use program.
According to data from the 2013 National Youth Tobacco Survey published by the Centers for Disease Control and Prevention, almost 23% of high school students surveyed had used a tobacco product within the previous 30 days, and 12.6% used two or more tobacco products. In addition, 46% of high school students and 17.7% of middle school students said they had used a tobacco product at least once in their lifetime. Three out of four high school smokers continue smoking into adulthood.
The Guardian, a publication in the UK, reported that "more than 40 drugs being paid for out of the government's Cancer Drugs Fund are to be reassessed to decide if they are worth the money." The fund was established in 2010 to help patients pay for drugs not covered by the UK National Health Service because, according to the UK's National Institute for Health and Care Excellence, their benefit doesn't justify the expense. If drugs are dropped from the fund, existing patients could still receive them, but new patients could not.
The Centers for Medicare and Medicaid Services announced that annual low-dose CT lung cancer screening may soon be reimbursed for certain people at high risk of the disease. The proposed plan would cover annual CT scans for people ages 55 to 74 with a smoking history of 30 pack-years who still smoke or who quit within the last 15 years; pack-years are determined by multiplying the number of packs smoked daily by the number of years a person has smoked. Private insurers will also be required to cover lung cancer screenings for this group of people starting in 2015.
Novartis announced that the FDA's Oncologic Drugs Advisory Committee did not recommend approval of the investigational compound panobinostat (LBH589), a pan-deacetylase inhibitor, for patients with previously treated multiple myeloma. The committee's opinion will be considered by the FDA in reviewing the drug's application, but the agency is not bound to follow the committee's guidance. The final decision regarding approval is made by the FDA.
Seattle, WA–based Dendreon Corporation filed for Chapter 11 bankruptcy protection in federal court in Delaware. The company says its operations will continue without interruption during the financial restructuring process. Dendreon makes the prostate cancer vaccine Provenge (sipuleucel-T).
While the incidence of colorectal cancer (CRC) in people age 50 and older has declined, the incidence among people ages 20 to 49 has increased, with the largest increase—1.99%—in people ages 20 to 34, according to a report published online by JAMA Surgery. "The increasing incidence of CRC among young adults is concerning and highlights the need to investigate potential causes and external influences such as a lack of screening and behavioral factors," the authors conclude.
Between 2002 and 2011, the costs associated with skin cancer in the United States increased five times as much as the costs associated with treatments for other cancers, according to a study from the Centers for Disease Control and Prevention published online in the American Journal of Preventive Medicine. The average annual cost for skin cancer treatment increased from $3.6 billion during 2002 to 2006, to $8.1 billion during 2007 to 2011, an increase of 126%. The average annual cost of treating all other cancers increased by 25% during the same period.
The levels of several toxic substances are elevated in cigar smokers and, for at least one potent cancer-causing agent, comparable to levels found in cigarette smokers, according to a study published in Cancer Epidemiology, Biomarkers & Prevention. The researchers found that cigar smokers had higher levels of cotinine, NNAL, cadmium, and lead than did nonsmokers, and the concentration of NNAL in daily cigar smokers is comparable to that of daily cigarette smokers. The findings suggest that smoking cigars may be as harmful as smoking cigarettes.
Beginning next year, Google will cover the cost of genetic tests from Foundation Medicine for all employees and their families navigating cancer treatment. The Cambridge, MA, company's tests spot mutations in solid tumors and blood cancers, and then identify targeted drugs that are approved for treating the cancer or that might be effective for off-label use. Patients are also informed about relevant clinical trials of investigational agents in which they may take part.
The FDA approved ramucirumab (Cyramza; Eli Lilly) for use in combination with paclitaxel for the treatment of patients with advanced gastric or gastroesophageal junction adenocarcinoma. The approval was based on a 665-patient trial in which median overall survival (OS) was 9.6 months for those who received the combination compared with 7.4 months for patients who received paclitaxel alone. Ramucirumab was approved in April as monotherapy for patients who failed first-line therapy.
Bristol-Myers Squibb announced that its PD-1 inhibitor Opdivo (nivolumab) led to a 1-year survival rate of 41% in patients with advanced squamous cell non–small cell lung cancer. The company said that the typical 1-year survival rate for such patients, whose cancer has progressed after treatment with at least two other therapies, ranges from 5.5% to 18%. The median OS in the 117-patient, phase II trial was 8.2 months.
Amgen said its experimental ovarian cancer drug trebananib did not show statistically significant improvement in OS, a secondary endpoint, in a phase III clinical trial. The OS for patients who received trebananib plus paclitaxel was 19.3 months compared with 18.3 months for patients who received a placebo plus paclitaxel. In an earlier analysis, patients who received trebananib showed a 34% reduction in the risk of disease progression or death, the study's primary endpoint.
Lexington, MA–based Promedior announced that the FDA granted Fast Track designation to PRM-151 for the treatment of myelofibrosis. PRM-151 is a recombinant form of an endogenous human protein, PTX-2, that acts as a monocyte/macrophage differentiation factor to prevent and potentially reverse fibrosis. The company will present data from a phase II clinical trial of the drug at the annual meeting of the American Society of Hematology in San Francisco, CA, on December 8.
MedImmune, the biologics research and development arm of AstraZeneca, agreed to purchase Munich, Germany's Definiens for initial consideration of $150 million. Definiens develops biomedical image and data-analysis software to accelerate and improve research in drug discovery, development, and diagnostics.
Boston, MA–based Dana-Farber Cancer Institute and Tokyo, Japan's Astellas Pharma announced a 3-year collaboration to research and develop small-molecule inhibitors of oncogenic KRAS. About 30% of all cancers harbor KRAS mutations, but despite more than two decades of research, KRAS has proven highly difficult to target, and no effective therapy currently exists.
According to a new report from the Centers for Disease Control and Prevention, about 8 million women ages 21 to 65 have not been screened for cervical cancer in the past 5 years. More than half of new cervical cancer cases occur among women who have never or rarely been screened. More than 12,000 women are diagnosed with cervical cancer every year; up to 93% of cervical cancers are preventable.
In collaboration with the NCI, FDA, industry members, and academic cancer center leaders, the Multiple Myeloma Research Foundation announced the start of the development of a Master Protocol that would enable several phase II clinical trials to take place within a single study design. Patients will be assigned to an appropriate experimental therapy based on an analysis of their genetic alterations. A similar Master Protocol study was launched earlier this year in lung cancer.
The FDA removed a full clinical hold on Geron Corporation's investigational new drug application for imetelstat, noting that the company's proposed plan to focus on high-risk myeloid malignancies such as myelofibrosis is acceptable. The company no longer plans to study the drug in patients with polycythemia vera or essential thrombocytopenia (ET). The FDA put a hold on the drug in February after ET patients in a phase II trial developed liver toxicity.
Approximately 100,000 people in Wales signed a petition calling for their government to establish a Cancer Drugs Fund similar to one in England. The fund allows patients to access drugs that are licensed but not recommended by the National Institute for Clinical Excellence due to their cost. Sky News reported that the Welsh Labour Government has resisted the idea.
The U.S. District Court for the District of Delaware ruled in favor of Pfizer in a lawsuit against Mylan Pharmaceuticals, upholding Pfizer's patent covering Sutent (sunitinib malate) capsules. Pfizer filed suit in 2010 after Mylan sought FDA approval to market a generic version of the drug, which is approved to treat advanced renal cell carcinoma and certain gastrointestinal and pancreatic tumors. Pfizer's patent expires in 2021.
The International Myeloma Working Group, which includes more than 180 researchers worldwide, updated the definition of multiple myeloma and the criteria that should be used to diagnose it. The new criteria, which include the use of validated biomarkers, were published in Lancet Oncology.
Merck announced that the FDA granted Breakthrough Therapy designation to Keytruda (pembrolizumab) for the treatment of EFGR-negative and ALK rearrangement–negative non–small cell lung cancer that has progressed while on or following platinum-based chemotherapy. The company's anti–PD-1 therapy has already been approved by the FDA for certain melanomas, and it is being studied in more than 30 types of cancer, both alone and in combination with other agents.
New Canadian guidelines recommend against using the prostate-specific antigen test to screen for prostate cancer based on evidence showing an increased risk of harm, such as false-positive results and unnecessary treatment, and little evidence of reduced mortality. The guidelines, published in the Canadian Medical Association Journal, are consistent with the recommendations of the U.S. Preventive Services Task Force and Cancer Council Australia; the UK does not have an organized screening program.
A noninvasive home screening test for colon cancer is now available with a prescription through the Mayo Clinic in Rochester, MN, the medical center where it was developed. The test, called Cologuard, checks for cancer-related DNA in stool. It is sold by Exact Sciences (Madison, WI) and will soon be available nationwide.
Investigators at Cleveland Clinic's Lerner Research Institute in Ohio will use IBM's "Watson" technology in genomic research to help oncologists deliver personalized medicine by uncovering new treatment options for a variety of cancers. Watson aims to find the "needle in the haystack" by identifying patterns in genome sequencing and medical data. Given Watson's ability to speedily review massive databases, researchers hope that more patients will have access to therapies tailored to their disease's DNA.
Citing their analysis of nearly 1,500 biotech and pharmaceutical companies in the United States and Europe, the executive recruitment services company Liftstream reports that the average board of directors is 90% male, and more than half of the industry's boardrooms do not include any women. Surveys from 530 leaders in the industry indicate that roughly the same proportion of men and women want to serve on a board, but nearly 60% of the men were contacted about such opportunities compared with about 16% of the women. The report, available at www.liftstream.com, suggests that CEOs and company boards should reexamine their recruiting and promotion practices, and evaluate training opportunities.
Concluding that "the transaction is no longer in the best interests of stockholders at the agreed upon valuation," AbbVie's Board of Directors has withdrawn its support for a proposed merger with the biopharmaceutical company Shire. The decision was spurred by a notice from the U.S. Department of Treasury regarding the tax implications of such a transaction. AbbVie will pay Shire a "break fee" of $1.635 billion.
Roche announced that it will invest about $3.15 billion (3 billion Swiss francs) over the next 10 years in the construction and renovation of state-of-the-art offices and laboratories at its Basel, Switzerland, site. The company said that many of the buildings on the site "no longer meet present-day requirements for modern, sustainable workplaces."
Reuters reported that a British petition signed by 29,000 people demands that Roche cut the price of its breast cancer drug Kadcyla (ado trastuzumab emtansine, or T-DM1). Britain's National Institute for Health and Care Excellence estimates that the drug costs about $145,000 per patient, which is too expensive for the nation's state-run health service.
The FDA granted Fast Track designation to Taiho Oncology's TAS-102, an investigational oral agent for the treatment of refractory metastatic colorectal cancer. Consisting of trifluridine and tipiracil hydrochloride, TAS-102 is an antineoplastic nucleoside analog that is incorporated into DNA and interferes with its function. The designation helps facilitate the development and expedite the review of new drugs intended to treat serious or life-threatening diseases.
The NIH announced awards totaling nearly $31 million to enhance diversity in the biomedical research workforce. The awards are part of a projected 5-year program to support more than 50 individuals, including those from backgrounds underrepresented in biomedical sciences, and partnering institutions in establishing a national consortium to develop, implement, and evaluate methods of encouraging people to pursue—and stick with—careers in biomedical research.
Genentech announced that it is partnering with Ames, IA–based NewLink Genetics to develop NLG919, an IDO pathway inhibitor akin to immune checkpoint inhibitors targeting CTLA-4, PD-1, and PD-L1. NewLink has also discovered tryptophan-2,3-dioxygenase specific inhibitors, potential anticancer compounds that could function alone or in combination with IDO inhibitors. NewLink will receive an up-front payment of $150 million and could receive $1 billion more for meeting certain milestones.
Knowing their individual risk for disease is not enough to persuade previously nonadherent patients to undergo recommended colorectal cancer screenings, according to a randomized, controlled trial published in Annals of Internal Medicine. Researchers had hypothesized that providing average-risk patients with a personalized genetic and environmental risk assessment would increase screening rates, but, after 6 months, there was no statistically significant difference in screening between those who received the personalized assessment and those who didn't. The researchers suggest that the findings debunk a common claim that greater knowledge of one's genetic makeup promotes healthier behaviors.
The Indiana University Melvin and Bren Simon Cancer Center in Indianapolis has once again been recognized as an NCI-designated cancer center. The designation will bring $7.8 million in federal grant money to the institution over the next 5 years, an increase of 20% from its previous award in 2008.
Millennium announced that the FDA approved Velcade (bortezomib) for use in previously untreated patients with mantle cell lymphoma (MCL), a subtype of non-Hodgkin lymphoma that is usually aggressive. According to the Cambridge, MA, company, Velcade is the first treatment to be approved for these patients in the United States. The drug was approved in 2006 for patients with relapsed or refractory MCL.
The FDA granted priority review to multiple cancer drugs: palbociclib (Pfizer), in combination with letrozole, as a first-line treatment for postmenopausal women with ER-positive, HER2-negative advanced breast cancer; lenvatinib (Eisai), a VEGF inhibitor, for the treatment of progressive radioactive iodine–refractory differentiated thyroid cancer; and blinatumomab (Amgen), an investigational bispecific T cell engager antibody construct, for the treatment of adults with Philadelphia-negative, relapsed or refractory B-precursor acute lymphoblastic leukemia. Decisions on the drugs are anticipated in April (palbociclib and lenvatinib) and May (blinatumomab) 2015.
Princeton, NJ–based Advaxis, a clinical stage biotechnology company developing cancer immunotherapies, announced preliminary data from a phase I/II study of its immunotherapeutic ADXS-HPV in anal cancer associated with human papillomavirus (HPV). All seven patients who have completed the treatment regimen experienced a "clinical complete response," the company said. The trial is a nonrandomized, open-label, multicenter study of 25 patients with anal cancer designed to assess the safety and effectiveness of ADXS-HPV in combination with standard chemotherapy and radiation.
The NIH announced $32 million in grants to fund the development of software and tools to help researchers mine large amounts of biomedical data, while protecting patient privacy, to improve human health. The awards are part of the agency's Big Data to Knowledge initiative, dubbed BD2K, which aims to invest nearly $656 million through 2020 in efforts to manage complex data sets.
To help speed the development and approval of drugs for certain breast cancers, the FDA released a guidance document for industry called "Pathological Complete Response in Neoadjuvant Treatment of High-Risk Early-Stage Breast Cancer: Use as an Endpoint to Support Accelerated Approval," which defines pathological complete response (pCR), discusses endpoints for neoadjuvant trials, and identifies suitable patient populations. However, the agency acknowledges that "important regulatory questions remain … regarding the use of pCR to support accelerated approval. A relationship between magnitude of improvement in pCR and improvement in long-term outcome has not been established at a trial level."
In conjunction with Ohio State University's (OSU) Comprehensive Cancer Center, named the Arthur G. James Cancer Hospital and Richard J. Solove Research Institute, OSU's Wexner Medical Center in Columbus has opened an emergency department dedicated solely to patients with cancer. The department will be staffed by oncologists and emergency medicine physicians who will treat an estimated 6,500 patients per year who seek emergency care for conditions related to cancer treatment such as severe dehydration, fever, and neutropenia.
The NCI launched the Exceptional Responders Initiative, a study that will investigate the molecular factors associated with dramatic responses to cancer treatment relative to responses in other patients receiving the same therapy. Researchers plan to examine tissue and clinical data and conduct gene sequencing in as many as 300 cases. Those interested in receiving more information or contributing to the study should email NCIExceptionalResponders@mail.nih.gov.
The U.S. Department of Health and Human Services added four substances to its 13th Report on Carcinogens, bringing the total number listed to 243. The report lists ortho-toluidine, used to make rubber chemicals, pesticides, and dyes, as a known human carcinogen. Three substances have been added as "reasonably anticipated" to be human carcinogens: 1-bromopropane, used as a cleaning solvent and adhesive; cumene, used to make phenol and acetone and also found in fuel products and tobacco smoke; and the wood preservative mixture pentachlorophenol.
The FDA granted Breakthrough Therapy designation to Cambridge, MA–based Ariad Pharmaceuticals' AP26113 for the treatment of ALK+ metastatic non–small cell lung cancer resistant to crizotinib (Xalkori; Pfizer). The designation, which helps expedite drug development and review, is based on results from an ongoing phase I/II trial that show sustained antitumor activity in patients with the disease, including those with brain metastases.
The FDA granted Orphan Drug designation to pegylated recombinant human hyaluronidase (PEGPH20) for the treatment of pancreatic cancer. Halozyme Therapeutics of San Diego, CA, is investigating PEGPH20 in combination with gemcitabine and nab-paclitaxel (Abraxane; Celgene) in a phase II study of patients with metastatic disease.
Tokyo, Japan–based Daiichi Sankyo agreed to buy Ambit Biosciences of San Diego, CA, for up to $410 million—$315 million in cash with additional payments contingent upon achieving certain milestones. Ambit's leading drug candidate is quizartinib (AC220), a potent and selective inhibitor of FLT3, a validated target in the treatment of acute myeloid leukemia.
An FDA advisory committee voted not to recommend approval of SonaCare Medical's high-intensity focused ultrasounddevice for the treatment of certain prostate cancers, citing the need for additional data. The device uses ultrasound energy to ablate tumors. The Charlotte, NC, company said that it will work with the FDA to find a "possible path forward."
To cut costs, Israel's Teva Pharmaceutical Industries announced that it will discontinue research and development in oncology and women's health, likely saving more than $150 million in 2015 and more than $200 million in 2016 and in 2017. The leading generic-drug maker will focus its resources on therapies for central nervous system and respiratory diseases and disorders.
In a new guidance document, the FDA recommends that manufacturers consider cybersecurity risks as part of the design and development of a medical device and submit documentation to the agency about the risks identified and controls in place to mitigate those risks. The guidance also recommends that manufacturers submit their plans for providing patches and updates to operating systems and medical software. Some medical devices can be vulnerable to security breaches, potentially affecting their safety and effectiveness, the FDA says.
A survey from GE Healthcare that included 10,000 adults from 10 countries found that 75% of respondents did not know that dense breast tissue increases a woman's risk of developing breast cancer. Respondents in the United States, Australia, the United Kingdom, and Japan were the least aware of this link (19%, 13%, 9%, and 2%, respectively), while respondents in Russia, Indonesia, and China were the most aware (60%, 58%, and 34%, respectively). In addition, less than half of all respondents could confidently name the most commons signs and symptoms of breast cancer.
The American Society of Clinical Oncology released its first-ever policy statement on cancer and obesity, underscoring that obesity is becoming a central challenge in cancer prevention and care and is projected to soon overtake tobacco use as the leading preventable cause of cancer in the United States. The statement, published in the Journal of Clinical Oncology, calls for increased education, research, and advocacy to reduce the toll of obesity as a cause of cancer and a complication in the care of patients.
Across Europe, more than 70% of young cancer specialists are showing signs of burnout, according to the results of a survey of 595 oncologists under age 40 presented at the Congress of the European Society for Medical Oncology in Madrid. There was no significant difference in burnout between men and women, but rates did vary across Europe. Burnout was highest in central Europe, affecting 84% of respondents, and lowest in northern Europe and the UK, where 52% of respondents were affected.
The FDA granted Orphan Drug designation to a MabVax Therapeutics bivalent vaccine that elicits an immune response against the two most common antigens on neuroblastoma cells: GD2 and GD3. The designation provides the San Diego, CA, company with substantial incentives such as market exclusivity, tax credits, fee exemptions, and closer collaboration with the FDA on the development of the vaccine. To that end, MabVax plans to launch a phase II trial of the vaccine next year.
The Los Angeles, CA–based biopharmaceutical company CytRx announced that its aldoxorubicin received Orphan Drug designation for three indications: glioblastoma multiforme, small cell lung cancer, and ovarian cancer. Aldoxorubicin, currently in phase II and III trials, combines the chemotherapeutic doxorubicin with a novel single-molecule linker that binds to albumin, a plentiful circulating protein soaked up by tumors. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released.
Also in the Journal of Clinical Oncology, researchers reported that the cost of treating cancer has risen dramatically in spite of a law passed in 2003 designed to control Medicare drug costs, including the cost of chemotherapy. Oncologists continued to prescribe expensive cancer drugs, they said, even after reimbursement for the drugs dropped in 2005. Researchers noted that the aggregate cost of cancer care rose by as much as 60% between 2003 and 2013.
Aliso Viejo, CA–based Ambry Genetics announced a research partnership aimed to recognize the significance of mutations and variants found by multigene testing for hereditary cancer. Participants in the Prospective Registry of MultiPlex Testing, or PROMPT, include researchers from Memorial Sloan Kettering Cancer Center (New York, NY), Mayo Clinic (Rochester, MN), Abramson Cancer Center of the University of Pennsylvania (Philadelphia), Dana-Farber Cancer Institute (Boston, MA), and other diagnostic testing laboratories. Together, they will create an online registry of patients who have undergone hereditary cancer testing, using the latest in next-generation gene sequencing technology, to learn more about genes on the panels that are associated with an increased risk of cancer.
FierceBiotech reports that Bristol-Myers Squibb plans to build a 2,500-employee campus in Lawrenceville, NJ. Slated to open in 2016, the 650,000-square-foot complex will consolidate two existing offices and provide room to grow.
Part of the World Health Organization, the International Agency for Research on Cancer (IARC) urges health authorities of countries with high rates of stomach cancer to include the disease in their national cancer-control programs and allocate more resources to control it, according to a new report. "Ignoring stomach cancer in the hope that it will soon disappear on its own is not a tenable health policy," says IARC Director Christopher Wild. In 2012, nearly 1 million cases were diagnosed worldwide.
With the new fiscal year (FY) starting next week but no new budget in place, the U.S. Congress approved a stopgap measure to fund the federal government from October 1 through December 11. The measure will continue funding for the NIH and NCI at their 2014 levels. Congress will work to finalize the FY2015 budget after the November elections.
Darmstadt, Germany's Merck KGaA announced that it will buy Sigma-Aldrich of St. Louis, MO, for $17 billion. Merck KGaA, which operates under the umbrella brand EMD in the United States and Canada, creates high-tech products in the pharmaceutical and chemical sectors. Sigma-Aldrich develops and manufactures a range of life science products, such as chemicals, biochemicals, and equipment for life science research, including genomic and proteomic research.
The parent company of Cambridge, MA–based Millennium Pharmaceuticals, Takeda Pharmaceutical, announced a company-wide reorganization that will include moving the Japanese company's global oncology efforts to Cambridge. Currently, Takeda's oncology work takes place at sites around the world. Takeda purchased Millennium in 2008.
Gilead Sciences announced that the European Commission granted marketing authorization for idelalisib (Zydelig) for chronic lymphocytic leukemia and follicular lymphoma. Idelalisib inhibits PI3K delta, a protein that is overexpressed in many B-cell malignancies and plays a role in proliferation and migration of cancer cells. The FDA approved the drug for both conditions in July.
In an initial public offering of stock, Cambridge, MA–based Tokai Pharmaceuticals raised more than $97 million to develop its prostate cancer drug galeterone. Galeterone works like a combination of enzalutamide (Xtandi), an androgen blocker, and abiraterone (Zytiga), which targets an enzyme required for androgen production, while also degrading the androgen receptor on tumor cells. Multiple phase II trials of the drug are under way.
In a deal worth up to $600 million, Boehringer Ingelheim and CureVac will collaborate on the development of CV9202, a novel investigational therapeutic mRNA vaccine for the treatment of lung cancer. Boehringer Ingelheim will begin clinical trials of the vaccine, in combination with afatinib (Gilotrif), in patients with EGFR-mutated non–small cell lung cancer (NSCLC), and in combination with chemoradiation therapy in patients with unresectable stage III NSCLC. Based in Ridgefield, CT, CureVac will initially receive $45 million, with additional payments for meeting certain milestones.
The FDA removed the clinical hold on patient enrollment and dosing of PEGPH20 in an ongoing phase Ib/II clinical trial evaluating the agent in combination with FOLFIRINOX chemotherapy in patients with metastatic pancreatic adenocarcinoma. PEGPH20 is an investigational pegylated form of Halozyme Therapeutics' proprietary recombinant human hyaluronidase under development for the systemic treatment of tumors that accumulate hyaluronan. Sponsored by the cancer research cooperative group SWOG, the trial aims to enroll 170 patients.
Scientists in Scotland were relieved when its residents voted last week to remain part of the UK instead of becoming an independent country, the New Scientist reported. Many worried that a split would compromise research funding. "This ensures that biomedical and clinical research—and Scotland's great achievements in delivering on this in the past—will continue unchecked," Margaret Frame, director of science at the Edinburgh Cancer Research Centre, told the publication.
Patients with KRAS-mutant pancreatic cancer who received the experimental drug Reolysin had a median progression-free survival (PFS) of 5.72 months compared with 4.11 months for those in the control arm—a 39% improvement, Calgary, Canada's Oncolytics Biotech announced. The randomized phase II study compared carboplatin, paclitaxel, and Reolysin, a proprietary formulation of the human reovirus, to carboplatin and paclitaxel alone.
According to a study in the Journal of the National Cancer Institute, 43% of women between 40 and 74 years old in the United States have mammographically dense breasts, which can make mammograms more difficult to read accurately. The finding provides a rationale for legislation requiring that women be notified if they have dense breasts, the researchers say. In addition, these women should work with their clinician to evaluate their risk of breast cancer and pursue additional screenings if appropriate.
Bethesda, MD–based Northwest Biotherapeutics announced that its DCVax-L, an immunotherapy under development for all malignant gliomas, is the first product to receive the UK's Promising Innovative Medicine (PIM) certification. Akin to the FDA's Breakthrough Therapy status, PIM designation is the initial step in the UK's Early Access to Medicines Scheme, which aims to speed patient access to medicines where there is an unmet medical need.
High-risk prostate cancer patients who receive radiation therapy and an 18-month course of androgen deprivation therapy (ADT) recover a normal testosterone level more quickly than those who receive a 36-month course of ADT, resulting in a better quality of life—without detriment to long-term outcomes, according to research presented at the American Society for Radiation Oncology's annual meeting in San Francisco, CA. The findings were based on data from 561 patients with high-risk prostate cancer enrolled in a multicenter, randomized phase III trial in Canada.
Nearly 300 organizations partnered in the Rally for Medical Research Hill Day in Washington, DC, to press senators and representatives to increase NIH funding and celebrate medical research.
Gilead Sciences said that its simtuzumab, in combination with chemotherapy, did not significantly improve PFS in previously untreated patients with advanced pancreatic cancer compared with a placebo plus chemotherapy in a phase II trial. Simtuzumab is also under study for use in colorectal cancer, myelofibrosis, idiopathic pulmonary fibrosis, and liver fibrosis.
Data released by the Department of Health in New York, NY, show that 16% of the city's adults smoked in 2013, up from 14% in 2010, the city's lowest recorded smoking rate. The increase is surprising, experts said, because smoking rates are generally falling across the country and because the city's former mayor, Michael Bloomberg, who served for 12 years beginning in 2002, oversaw efforts to decrease smoking by banning it in public places and increasing taxes on tobacco products.
To mark its 50th anniversary, the American Society of Clinical Oncology announced the "Top 5 Advances in 50 Years of Modern Oncology," based on more than 2,000 votes from around the world: a four-drug chemotherapy regimen called MOPP that cures advanced Hodgkin lymphoma; the approval of a human papillomavirus vaccine to prevent cervical cancer; the approval of imatinib (Gleevec; Novartis) for the treatment of chronic myeloid leukemia; a three-drug chemotherapy regimen called PVB that cures men with testicular cancer; and the approval of antinausea drugs to improve cancer patients' quality of life.
Pharmacy chain CVS stopped selling tobacco a month earlier than originally planned and changed its name from CVS Caremark to CVS Health to better reflect its growing commitment to customer health. Displays of cigarettes and cigars have been replaced with nicotine gum and other smoking cessation products. In February, the company had announced it would end tobacco sales by Oct. 1.
The American Society of Clinical Oncology issued a new clinical practice guideline for women with HER2-negative advanced breast cancer. It says that chemotherapies should be given sequentially, rather than in combination, to reduce side effects, and that no targeted agents should be used in addition to, or as a replacement for, chemotherapy. Because there is no one optimal chemotherapy, the guideline recommends that doctors and patients make treatment choices together, considering prior therapies, side effects, dosing schedule, and other medical conditions.
General Electric received approval from the FDA to sell its 3-D breast-imaging devices in the United States. Also called tomosynthesis, 3-D mammography combines X-rays taken from multiple angles to create more-detailed images than traditional mammograms. Until now, Hologic, which received FDA approval in 2001, has been the only company allowed to sell the machines in the United States.
South San Francisco, CA–based Exelixis announced that its cancer drug cabozantinib did not improve survival in men with advanced prostate cancer who were no longer responding to docetaxel. Median survival for the 960 men enrolled in the phase III trial was 11 months for those who received cabozantinib versus 9.8 months for those who received prednisone, which was not statistically significant. Given the negative results, the company will eliminate 70% of its staff, or 160 employees, according to The Street.
The Cancer Drugs Fund will get an extra £160 million ($265 million) and be extended to 2016 to help patients in England receive cancer medications that the country's National Health Service would not ordinarily cover. The infusion of cash will bring the fund, which has helped more than 55,000 cancer patients since it was set up 4 years ago, to £280 million a year.
In response to revised study protocols, the FDA released a partial hold on patient enrollment in three phase I clinical trials of OncoMed Pharmaceuticals' experimental cancer drug vantictumab, the Redwood City, CA, company announced. The hold occurred on July 1, after mild-to-moderate bone-related adverse events were observed in patients. Vantictumab selectively targets Frizzled receptors, activators of Wnt signaling.
The Leukemia and Lymphoma Society partnered with OncoPep to advance an experimental vaccine, PVX-410, designed to treat patients with smoldering multiple myeloma, an asymptomatic stage of myeloma. The organization has committed to an equity investment of $690,000 to support clinical development of the therapy, which was granted Orphan Drug designation by the FDA in 2013. To date, 12 patients have been treated with the vaccine in an ongoing phase I/II clinical trial.
NCI's Clinical Proteomic Tumor Analysis Consortium and the NVIDIA Foundation announced that they will collectively award up to $2 million for the development of data-intensive scientific tools to find new ways to treat cancer. The organizations are seeking proposals for "projects that leverage computation on integrating large-scale, publicly available omics data sets to dramatically impact the battle against cancer and reduce the time it takes for research outcomes to be used effectively in a clinical environment." Applications are due October 8.
Santa Monica, CA–based Kite Pharma, Inc., announced that administration of KTE-C19, an anti-CD19 chimeric antigen receptor T-cell therapy, yielded an overall response rate of 92% in 13 evaluable patients with B-cell malignancies. Eight patients in the phase I-IIa study experienced a complete remission and four experienced a partial remission. The findings were published in the Journal of Clinical Oncology.
Although it backs the battery-powered devices as a last resort to help smokers quit, the American Heart Association issued a policy statement urging government to more heavily regulate electronic cigarettes. E-cigarettes, as they are called, are less toxic than traditional cigarettes, but little evidence exists to support claims that they aid smoking cessation, and concerns abound that their candy and fruit flavorings will attract youth. A day later, the World Health Organization also called for increased regulation, such as bans on advertising, indoor use of e-cigarettes, and the sale of the devices to anyone under age 18, in advance of an international tobacco-control meeting scheduled for October.
Illumina announced that it will partner with AstraZeneca, Janssen Biotech, and Sanofi to develop a universal next-generation sequencing–based oncology test system to detect and measure multiple variants simultaneously. The system will be used for clinical trials of targeted cancer therapies with a goal of developing and commercializing a multigene panel to select the best therapy for each patient.
The Herbert Irving Comprehensive Cancer Center of Columbia University Medical Center and New York-Presbyterian Hospital earned an $18 million, 5-year cancer center support grant from the NCI. The grant renews the New York City center's status as an NCI-designated comprehensive cancer center.
GE Healthcare Life Sciences announced that it will open its new U.S. headquarters in Marlborough, MA, investing $21 million in a currently unoccupied site. Slated to open in the spring, the 160,000 square-foot facility will employ 500 workers and create 220 new jobs. The company's global headquarters will remain in Amersham, UK.
According to the London, UK, research and consulting firm GlobalData, the market for drugs to treat B-cell non-Hodgkin lymphoma will experience moderate growth, from $4.59 billion in 2013 to $5.41 billion by 2018, across the United States, France, Italy, Germany, Spain, and the UK. The market will undergo a dramatic shift away from chemotherapeutics to targeted B-cell receptor inhibitors such as ibrutinib (Imbruvica; Pharmacyclics and Janssen) and idelalisib (Zydelig; Gilead), the firm says.
The NIH is challenging science innovators to compete for prizes totaling up to $500,000 by developing new ways to track the health status of a single cell in complex tissue over time. The Follow that Cell Challenge seeks tools that would, for example, monitor a cell in the process of becoming cancerous or how a cell responds to treatment.
Having completed a 14-month engagement at the Smithsonian's National Museum of Natural History in Washington, DC, the exhibition "Genome: Unlocking Life's Code" is on the move to San Diego, CA, for the first of eight planned stops throughout North America. A collaboration between the museum and the National Human Genome Research Institute, part of the NIH, the exhibition's eye-catching models and interactive displays that have already attracted 3 million visitors. It opens in San Diego on September 24.
Baseball pitching great Curt Schilling announced that he has been treated for an oral squamous cell carcinoma that he "unquestionably" believes was caused by decades of chewing tobacco use, saying he wished he "could go back and never have dipped." Earlier this year, Hall of Famer Tony Gwynn died of salivary gland cancer, which he also attributed to chewing tobacco. Cancer prevention advocates say they hope Schilling's and Gwynn's experiences will discourage people, particularly young baseball fans, from using any type of tobacco.
The FDA approved bevacizumab (Avastin; Genentech) in combination with chemotherapy to treat women with persistent, recurrent, or metastatic cervical cancer, making it the first drug approved for these patients since 2006. The angiogenesis inhibitor is also approved to treat various forms of brain, colon, kidney, and lung cancers.
Agios Pharmaceuticals of Cambridge, MA, announced that the FDA has granted Fast Track designation to AG-221 for the treatment of patients with acute myelogenous leukemia. AG-221 is a first-in-class, oral, selective, potent IDH2 inhibitor being evaluated in a phase I clinical trial. The designation, given to medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs, means that Agios will have frequent interactions with the FDA to expedite the drug's development.
Pharmaceutical giant Pfizer announced that it has submitted an application to the FDA for approval of palbociclib, in combination with letrozole, as a first-line systemic therapy for postmenopausal women with ER+, HER2– advanced or metastatic breast cancer. Palbociclib is an investigational oral targeted agent that selectively inhibits cyclin-dependent kinases 4 and 6. The submission is based upon the results of a phase II study in which patients who took palbociclib and letrozole had progression-free survival of 20.2 months compared with 10.2 months in patients who took letrozole alone.
Ohio's Cleveland Clinic unveiled plans to build a $276 million, seven-story cancer center, which will bring all cancer-related services, including imaging, under one roof. The additional space will also allow the institution to increase the number of patients it can accommodate in clinical trials. Groundbreaking is slated for the end of September, with construction to be completed in 2017.
The University of Texas at Austin announced that the Livestrong Foundation will contribute $50 million to the school's new Dell Medical School to create the Livestrong Cancer Institutes. The gift is the largest ever from the foundation, which was established in 1997 by cyclist Lance Armstrong. The medical school, which will welcome its first class in the fall of 2016, was named for Michael and Susan Dell, who had earlier donated $50 million.
The NIH launched ALCHEMIST (Adjuvant Lung Cancer Enrichment Marker Identification and Sequencing Trials) to identify patients with early-stage lung tumors that harbor genetic changes in ALK and EGFR and evaluate whether drugs targeted against those changes can improve survival. About 6,000 to 8,000 patients will be screened for the alterations, which are present in a total of about 15% of lung cancers. Those with the genetic changes will then be eligible for one of the two treatment trials; all screened participants, regardless of the marker status of their tumor, will be followed for 5 years.
A long-term study reported in the journal Pediatrics shows that the human papillomavirus (HPV) vaccine appears to protect against the sexually transmitted virus for at least 8 years. HPV causes genital warts, as well as most cases of cervical cancer and certain head and neck cancers. Boys and girls ages 9 to 15 who received the vaccine as part of the study still had antibodies against HPV 8 years later, and none developed any conditions or diseases related to the virus.
The FDA approved Exact Sciences' Cologuard, the first stool-based colorectal screening test that detects the presence of red blood cells and DNA mutations that may indicate the existence of cancer or a precursor to cancer. The Centers for Medicare & Medicaid Services proposed covering the test once every 3 years for people ages 50 to 85 who are asymptomatic and at average risk of colorectal cancer.
The FDA also approved the use of bortezomib (Velcade; Millennium) for the retreatment of adults with multiple myeloma who previously responded to the drug but relapsed at least 6 months after completing the therapy. The approval was based on a phase II, single-arm study of 130 such patients that showed an overall response rate of 38.5% and a median duration of response of 6.5 months. Bortezomib was approved in 2005 for patients with multiple myeloma who had received at least one prior treatment and in 2003 for patients with more-refractory disease.
More than 4.3 million women with limited access to health care received breast and cervical cancer screening and diagnostic services in the first 20 years of a program of the Centers for Disease Control and Prevention (CDC)—the National Breast and Cervical Cancer Early Detection Program. Through the program, 56,662 breast cancers, 3,206 cervical cancers, and 152,470 precancerous cervical lesions were detected, and more than 90% of these women received timely follow-up care. A CDC report on the program is scheduled to be published on August 15 in a supplement to the journal Cancer.
Seattle, WA–based CTI BioPharma announced that its investigational drug pacritinib received Fast Track designation from the FDA for the treatment of intermediate- and high-risk myelofibrosis. An oral tyrosine kinase inhibitor with activity against both JAK2 and FLT3, pacritinib is being evaluated in two phase III trials.
Attorneys general from 29 states urged the FDA to strengthen its proposed regulations on electronic cigarettes, or e-cigarettes, saying that the rules proposed by the agency in April fail to address multiple concerns. For example, they want the regulations to prohibit the use of flavorings in e-cigarettes, as has been done in traditional cigarettes, because flavors like chocolate, grape, and cherry appeal to children. They also called for greater restrictions on the advertising and marketing of the devices.
Mirati Therapeutics announced that the FDA granted Orphan Drug designation to mocetinostat, a spectrum-selective HDAC inhibitor, for diffuse large B-cell lymphoma. In June, San Diego, CA–based Mirati announced that mocetinostat received Orphan Drug designation as a treatment for myelodysplastic syndrome. The company plans to seek the designation for bladder cancer with specific genetic alterations.
Cancer Research UK (CRUK) called upon scientists, doctors, and nurses to get more involved in Wikipedia to help ensure that the free, online encyclopedia has the most up-to-date and accurate information on cancer. "We feel it's our responsibility to make sure that cancer information online is as good as it can be, to remove uncertainty, reduce fear and worry, and to help people take their next steps after they, their family or friends hear the phrase, 'it's cancer,'" says Henry Scowcroft, CRUK's news and multimedia manager.
The FDA issued final guidance on the development, review, and approval or clearance of companion diagnostics, which are tests used to identify patients who will likely benefit from treatment with a particular drug. The tests are commonly used to detect certain types of gene-based cancers.
On July 31, the FDA also notified Congress of its intent to publish a proposed risk-based oversight framework for laboratory-developed tests (LDT), which are designed, manufactured, and used within a single laboratory; this draft guidance document will not be published for at least 60 days. The agency already oversees direct-to-consumer tests regardless of whether they are LDTs or traditional diagnostics.
Amgen and its subsidiary, Onyx Pharmaceuticals, announced that a planned interim analysis demonstrated that a phase III trial of carfilzomib (Kyprolis) met its primary endpoint of progression-free survival in patients with multiple myeloma. Those treated with carfilzomib in combination in lenalidomide (Revlimid) and low-dose dexamethasone lived a median of 26.3 months without their disease worsening, compared with 17.6 months for patients treated with lenalidomide and low-dose dexamethasone alone. Data for overall survival are not yet mature.
Despite the success of carfilzomib and increasing sales of its other products, Amgen said it plans to cut its global workforce by 12% to 15%, up to about 2,900 jobs. The layoffs will begin later this year and run through 2015. Most of the workforce reductions will occur in the United States.
The European Medicines Agency and the FDA have granted Orphan Drug Designation to AbbVie's investigational compound ABT-414, an anti-epidermal growth factor receptor antibody–drug conjugate, the company announced. ABT-414 is being evaluated for safety and efficacy in patients with glioblastoma multiforme.
Incyte Corporation announced that the FDA granted priority review to ruxolitinib (Jakafi) as a potential treatment for patients with polycythemia vera who have had an inadequate response to, or are intolerant to, hydroxyurea. The FDA is expected to make a decision on the JAK1/JAK2 inhibitor by December 5. The drug is already approved for the treatment of myelofibrosis.
A new guideline from the U.S. Multi-Society Task Force on Colorectal Cancer recommends genetic testing of all tumors from patients newly diagnosed with colorectal cancer for evidence of mismatch repair deficiency to help identify those with Lynch syndrome. Subsequent germline genetic testing can confirm the diagnosis and guide the care of the patient and any family members. Lynch syndrome is the most common cause of inherited colorectal cancer.
Johnson & Johnson's Ethicon initiated a worldwide voluntary market withdrawal of its morcellators, devices used during minimally invasive hysterectomy for symptomatic uterine fibroids and myomectomy. The company stopped marketing the devices in April amid concerns that the devices might spread an unsuspected cancer. The FDA is currently evaluating the risks and potential benefits of the use of laparoscopic power morcellators.
UK-based Wellcome Trust announced that it will invest £27 million in a state-of-the-art genome-sequencing hub for Genomics England, the government's project to decipher 100,000 complete genetic codes. The funding will allow Genomics England to become part of the Wellcome Trust Genomics Campus in Hinxton, which is also home to the Sanger Institute, the European Bioinformatics Institute, and several small biotech companies.
The University of Texas MD Anderson Cancer Center in Houston has announced a partnership with Hospital Israelita Albert Einstein to pioneer multidisciplinary patient care in Brazil. The 489-bed private hospital in São Paulo will be the first clinical extension of MD Anderson in Latin America.
The Centers for Disease Control and Prevention (CDC) called upon healthcare providers to strongly recommend the human papillomavirus (HPV) vaccine at the same visit they recommend the Tdap and meningococcal vaccines for preteens, giving the recommendation for all of the shots the same weight. Only 64.4% of parents of girls and 41.6% of parents of boys received a recommendation for the HPV vaccine, according to the CDC, and only 57.3% of girls and 34.6% of boys have received at least one dose of the trivalent vaccine, compared with about 86% of teens who have received the Tdap vaccine.
The FDA expanded the approved uses for ibrutinib (Imbruvica; Pharmacyclics and Janssen Biotech) to include the treatment of patients with chronic lymphocytic leukemia (CLL) who carry a deletion in chromosome 17 associated with poor responses to standard treatment. The agency also approved new labeling for ibrutinib in response to new progression-free and overall survival data confirming its clinical benefit in CLL. In February, the drug received accelerated approval to treat CLL based on the overall response rate.
Gilead's idelalisib (Zydelig) and ibrutinib were both recommended for approval by the European Medicines Agency's Committee for Medicinal Products for Human Use. The drugs are recommended for use in treating adult patients with CLL who have received at least one prior therapy, as well as for first-line treatment of patients who are not candidates for chemoimmunotherapy. The drugs are also recommended for use in certain lymphomas.
Also in Europe, Roche's obinutuzumab (Gazyva) was approved for patients with previously untreated CLL, in combination with chlorambucil chemotherapy. Obinutuzumab is a glycoengineered anti-CD20 monoclonal antibody.
AstraZeneca partnered with Roche and Qiagen to develop two separate companion diagnostic tests to identify patients likely to benefit from its lung cancer drugs. Roche will develop a test to identify EGFR mutations in tumors and plasma from patients with non–small cell lung cancer (NSCLC) to optimize clinical development of AZD9291 for patients who are resistant to first-generation tyrosine kinase inhibitors. Qiagen will develop a test to analyze plasma samples to determine EGFR mutation status in patients with NSCLC to guide treatment with gefitinib (Iressa) when tumor tissue is not available.
The Cambridge, MA, company Foundation Medicine has joined the Addario Lung Cancer Medical Institute and the Bonnie J. Addario Lung Cancer Foundation to find better treatments for young adults with lung cancer and tailor therapies to them. Of the 224,000 people expected to be diagnosed with lung cancer this year, about 3,700 will be under age 45, according to the American Cancer Society. These younger patients often lack typical signs and symptoms of the disease, which may take a different course.
Acting Surgeon General Boris Lushniak, MD, MPH, issued a call to action to prevent skin cancer, saying that the disease is "a major public health problem." Noting that nearly 5 million people are treated for skin cancer in the United States every year at a cost of $8.1 billion, he suggested several strategies to reduce the risk of the disease, including increasing shaded areas outdoors, instituting better educational programs, greater enforcement of indoor tanning laws, and more behavioral research.
A St. Jude Children's Research Hospital study found that 73% percent of adult survivors of childhood cancer more than doubled their risk of developing metabolic syndrome and related health problems by failing to follow a heart-healthy lifestyle. The results were published in the journal Cancer. Metabolic syndrome is associated with greater odds of developing heart disease, diabetes, stroke, and other potentially fatal health problems.
The FDA granted traditional approval to idelalisib (Zydelig; Gilead) for use with rituximab (Rituxan; Genentech) for the treatment of relapsed chronic lymphocytic leukemia. The agency also granted accelerated approval to the drug for the treatment of relapsed follicular B-cell non-Hodgkin lymphoma and relapsed small lymphocytic lymphoma, another type of non-Hodgkin lymphoma.
A week after five U.S. senators wrote to the Office and Management and Budget (OMB) seeking the release of draft guidance on laboratory-developed tests (LDT) from the FDA, 20 lab directors penned a letter to the OMB "in opposition to FDA regulation of LDTs." They argued that LDTs are vital to the practice of medicine and "often created in response to unmet clinical needs"; that LDTs are not medical devices and thus are not subject to FDA regulation; that LDTs are already highly regulated; and that FDA regulation would "stifle innovation" and "be contrary to the public health." In their letter, the senators wrote that the draft guidance "has languished" at the OMB for years.
Los Angeles, CA–based Puma Biotechnology announced that women with early-stage HER2+ breast cancer who took neratinib (PB272) for adjuvant treatment of their disease as part of a phase III clinical trial experienced 33% improvement in disease-free survival compared with those who took a placebo. The trial included 2,821 patients in 41 countries who had had surgery and adjuvant treatment with trastuzumab who then received extended adjuvant treatment with neratinib or a placebo for 1 year. Based on the study's findings, which will be reported in detail at an upcoming scientific meeting, Puma plans to file for FDA approval of neratinib in the first half of 2015.
The FDA granted its Breakthrough Therapy designation to Aduro BioTech's pancreatic cancer combination treatment consisting of the immunotherapies CRS-207 and GVAX Pancreas. The Berkeley, CA, company noted that the designation was based a phase II trial in which the median overall survival of patients who received the combination treatment was 6.1 months compared with 3.9 months for those who received GVAX Pancreas alone.
GlaxoSmithKline halted a phase III trial of its MEK inhibitor trametinib (Mekinist) in combination with its BRAF inhibitor dabrafenib (Tafinlar) based on an interim analysis showing an overall survival benefit. The trial was comparing the combination to vemurafenib (Zelboraf; Genentech) in patients with BRAF V600E or V600K mutation–positive unresectable or metastatic cutaneous melanoma. Patients taking vemurafenib will be allowed to the switch to the trametinib–dabrafenib combination.
The FDA granted Priority Review to Genentech's bevacizumab (Avastin) plus chemotherapy for certain cervical and ovarian cancers based on data from two separate phase III trials. In one trial, women with persistent, recurrent, or metastatic cervical cancer who received bevacizumab plus chemotherapy experienced a 29% reduction in the risk of death compared with those who received chemotherapy alone. In the other, women with platinum-resistant, recurrent epithelial ovarian, primary peritoneal, or fallopian tube cancer who took bevacizumab plus chemotherapy reduced their risk of the disease worsening by 52% compared with those taking chemotherapy alone.
Eli Lilly and Company and Immunocore Limited announced that they will collaborate to discover and develop novel T cell–based cancer therapies using Immunocore's ImmTAC technology. The deal will earn UK-based Immunocore $25 million for each candidate therapy developed, the companies said.
NIH is piloting longer grant awards "to provide more stable support for investigators at all career stages," wrote Sally Rockey, PhD, deputy director for extramural research at NIH, and NIH Director Francis Collins, MD, PhD, on Rockey's "Rock Talk" blog. To that end, NCI is launching the Outstanding Investigator Award, "which will provide long-term support to investigators who have extraordinary records of cancer research productivity and who propose to conduct exceptional research." Applicants can request up to $600,000 a year for up to 7 years.
Roche announced that its phase III study of cobimetinib met its primary endpoint. An investigational MEK inhibitor, cobimetinib used in combination with Roche's BRAF inhibitor vemurafenib (Zelboraf) helped patients with previously untreated BRAF V600 mutation–positive advanced melanoma live significantly longer without their disease worsening compared with vemurafenib alone. The company said data will be presented at an upcoming medical meeting and submitted to the FDA for marketing approval.
The levonorgestrel-releasing intrauterine system, or progesterone-releasing IUD, may be associated with a higher-than-expected incidence of breast cancer, according to a study of 93,843 Finnish women ages 30 to 49 who used the device to treat heavy menstrual bleeding from 1994 to 2007. Published in Obstetrics and Gynecology, the study found that the device did not increase the risk of uterine, ovarian, pancreatic, or lung cancers, but the number of new breast cancer cases was 19% higher in women who used the device than in the general population.
The American Thoracic Society, American College of Chest Physicians, and other organizations in the Forum of International Respiratory Societies issued a position statement taking a dim view of electronic cigarettes. "As a precaution," the statement reads, "electronic nicotine-delivery devices should be restricted or banned until more information about their safety is available. If they are allowed, they should be closely regulated as medicines or tobacco products."
MabVax Therapeutics, Inc., and Telik, Inc., finalized their merger, with MabVax becoming a wholly owned subsidiary of Telik. Located in Palo Alto, CA, Telik is a clinical-stage drug development company focused on discovering and developing small-molecule drugs for cancer. MabVax focuses on the development of vaccines and antibody-based therapies for cancer.
According to research published in the Journal of Clinical Oncology, patients with colorectal cancer who had the highest levels of vitamin D in their blood were half as likely to die of the disease as those with the lowest levels. Researchers tested blood samples from nearly 1,600 patients following surgery and found that patients with large tumors that had not yet metastasized derived the greatest benefit. The team now plans to conduct a study to determine whether taking vitamin D supplements in combination with chemotherapy can improve survival rates among patients with colorectal cancer.
The V Foundation for Cancer Research and Stand Up To Cancer will collaborate on grants for research scholars working within an emerging scientific field known as convergence, a model of research meant to spur innovation in combatting cancer. Announced at the 2014 ESPYs, a sports awards ceremony televised on ESPN, the grants will support collaborative cancer research among different fields of study, such as engineering, physical sciences, and life sciences. Stand Up To Cancer is investing $4 million in the grants program.
The FDA granted accelerated approval to Spectrum Pharmaceuticals' Beleodaq (belinostat) for patients with peripheral T-cell lymphoma, a subtype of non-Hodgkin lymphoma, who have relapsed or not responded to prior treatment. The approval was based on the results of a multicenter, single-arm trial of 120 evaluable patients in which the primary endpoint was overall response rate (ORR). The ORR was 25.8%, with complete and partial response rates of 10.8% and 15.0%, respectively; the media duration of response was 8.4 months.
Novartis announced that the FDA designated its CTL019 a "Breakthrough Therapy" for the treatment of relapsed or refractory acute myeloid leukemia (AML). CTL019 is an investigational chimeric antigen receptor therapy that uses a patient's own T cells to target CD19-expressing cancer cells.
CASI Pharmaceuticals of Rockville, MD, announced that the FDA granted Orphan Drug Designation to ENMD-2076, an orally active Aurora A/angiogenic kinase inhibitor for the treatment of hepatocellular carcinoma. ENMD-2076 also targets the VEGFR, Flt-3, and FGFR3 kinases. The drug previously received Orphan Drug Designation for the treatment of ovarian cancer, multiple myeloma, and AML.
Results from a phase II study published in Blood showed that previously untreated patients with AML who were treated with volasertib combined with low-dose cytarabine (LDAC) had a median overall survival of 8.0 months compared with 5.2 months in patients treated with LDAC alone. The response rate more than doubled for patients receiving volasertib plus LDAC versus LDAC alone (31% versus 13.3%). Volasertib (Boehringer Ingelheim) is an investigational compound that inhibits Polo-like kinases, resulting in apoptosis.
The Japanese Ministry of Health, Labor, and Welfare approved Roche's alectinib for patients with ALK+ non–small cell lung cancer whose tumors are advanced, recurrent, or inoperable. The approval was based on results from a phase II study in which more than 90% of 46 patients responded to the drug. Progression-free survival at 12 months was 83%.
Japan also approved the PD-1 inhibitor nivolumab (Ono Pharmaceuticals) for the treatment of inoperable melanoma. The drug is licensed to Bristol-Myers Squibb outside of Japan, Korea, and Taiwan. Until now, the only standard drug therapy for patients in Japan with advanced melanoma has been the chemotherapy dacarbazine.
New York's Cold Spring Harbor Laboratory received a $50 million gift from the Jim and Marilyn Simons Foundation to establish a center focused on using quantitative biology to interpret genomic research data from a variety of diseases.
The European commission granted marketing authorization for GlaxoSmithKline's (GSK) Mekinist (trametinib) for adult patients with unresectable or metastatic melanoma with the BRAF V600 mutation. The Commission also granted marketing authorization for a new indication for Arzerra (ofatumumab; GSK and Genmab). The human monoclonal antibody against CD20, in combination with chlorambucil or bendamustine, can be used for the treatment of patients with chronic lymphocytic leukemia who have not received prior therapy or are not eligible for fludarabine-based therapy.
Hawaii became the 10th state to ban indoor tanning for people under age 18, joining Vermont, California, Illinois, Louisiana, Minnesota, Oregon, Nevada, Texas, and Washington. The law took effect immediately.
Swiss drugmaker Roche announced that Genentech, a unit of Roche based in San Francisco, CA, will acquire Seragon Pharmaceuticals of San Diego, CA, for $725 million, plus contingent payments of up to $1 billion if Seragon's products achieve certain milestones. With the acquisition, Genentech will obtain rights to Seragon's entire portfolio of investigational, next-generation oral selective estrogen receptor degraders for the treatment of hormone receptor–positive breast cancer. Seragon's leading drug, ARN-810, is in phase I clinical trials.
Lexington, MA-based Agenus announced that patients with glioblastoma multiforme who received its Prophage autologous cancer vaccine in addition to standard treatment with radiation and temozolomide had a median overall survival of about 24 months, compared with about 12 months for patients who received standard treatment alone. In addition, patients in the multi-institutional, open-label phase II trial who received the vaccine had a median progression-free survival of about 18 months, about two to three times longer than patients in the control group. The company noted that the response to Prophage seemed to be more pronounced in patients with less expression of the checkpoint ligand PD-L1 on their white blood cells.
At the annual meeting of the European Society of Human Reproduction and Embryology, researchers reported that there is "little evidence" that the use of conventional fertility hormones to treat infertility increases the long-term risk of breast or gynecologic cancers, according to the results of a 30-year follow-up study. However, extended use of clomiphene citrate (for 12 or more cycles) was associated with a higher risk of breast cancer.
According to results of a phase III study published in The Journal of the American Medical Association, everolimus (Afinitor; Novartis) does not significantly improve overall survival in patients with advanced hepatocellular carcinoma whose disease progressed following treatment with sorafenib (Nexavar; Bayer/Onyx). Median overall survival for patients in the everolimus group was 7.6 months, compared with 7.3 months in the placebo group. Median time to disease progression was 3.0 months with everolimus and 2.6 months with placebo.
The NCI renewed Roswell Park Cancer Institute's (RPCI) designation as a comprehensive cancer center. Located in Buffalo, NY, the center has held the distinction for 40 years. The award will bring $19 million in federal grant money to RPCI over the next 5 years.
The FDA granted Breakthrough Therapy designation to Amgen's blinatumomab (AMG 103) for adults with Philadelphia chromosome–negative relapsed/refractory B-precursor acute lymphoblastic leukemia, which will help expedite development and review of the drug. Blinatumomab is an investigational bispecific T-cell engager antibody designed to direct T cells against target cells expressing CD19. Bispecific T-cell engager antibodies engage two different targets simultaneously.
Rhode Island Governor Lincoln Chafee signed legislation that bans the sale of e-cigarettes to minors. The legislation also prohibits anyone in the state under age 18 from using the devices.
The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 11 to 2 that current evidence from clinical studies does not support accelerated approval of olaparib as a maintenance treatment for women with platinum-sensitive relapsed ovarian cancer who have the germline BRCA mutation, and who are in complete or partial response to platinum-based chemotherapy. The FDA, which is expected to issue a decision by October 3, does not need to follow ODAC's advice but generally does so. Drug maker AstraZeneca will continue to evaluate olaparib in a phase III trial.
Bisphenol A (BPA), a chemical commonly used in plastics that disrupts hormones, appears to increase the proliferation of breast cancer cells, Duke Medicine researchers reported at the annual meeting of the International Society of Endocrinology and the Endocrine Society in Chicago, IL, on June 23. The researchers also found that levels of BPA typically observed in human blood reduced the efficacy of FDA-approved drugs used to treat breast cancer, notably lapatinib (Tykerb; GlaxoSmithKline).
The FDA placed brentuximab vedotin (Adcetris) on its quarterly list of drugs to monitor because it may lead to hepatotoxicity. The Seattle Genetics drug was approved in 2011 to treat Hodgkin lymphoma and systemic anaplastic large cell lymphoma. Adding the drug to the watch list means that the FDA will investigate whether there is a causal link and, if so, the agency could require revisions to the drug's label.
The Centers for Disease Control and Prevention (CDC) announced that it will launch a new series of hard-hitting anti-tobacco ads—part of its "Tips from Formers Smokers" campaign—that will run nationwide for nine weeks beginning July 7 on television, radio, and billboards, as well as online and in theaters, magazines, and newspapers. The educational campaign has helped hundreds of thousands of smokers quit since it began in 2012, according to the CDC.
The Times of India reported that the Indian Patent Office denied a patent for Celgene's anticancer drug Abraxane (nab-paclitaxel), claiming that it lacks "an inventive step" Under Indian law, patents cannot be granted to new forms of known substances unless they show enhanced efficacy over the known substance. The patent was initially denied in 2009, but the decision was appealed to the Intellectual Property Appellate Board, which directed the Patent Office to reconsider its decision earlier this year.
After a National Academy of Sciences report raised significant concerns about its design, management, and oversight, the National Children's Study was put on hold by NIH Director Francis Collins, MD, PhD. The study, requested by Congress as part of the Children's Health Act of 2000, is envisioned to be a longitudinal, observational study of 100,000 children from the womb to age 21 that will examine the effects of environmental and biological factors on children's health, growth, and development.
The FDA warned that the intravenous chemotherapy drug docetaxel contains ethanol, which may cause patients to experience intoxication or feel drunk during or after treatment. Labels of docetaxel products will be revised to note the risk, but in the meantime, health care professionals should consider the drug's alcohol content when prescribing or administering the drug to patients. Docetaxel is used to treat certain breast, prostate, stomach, lung, and head and neck cancers.
The Centers for Disease Control and Prevention (CDC) reported that cigarette smoking rates among high school students have dropped to the lowest levels since the National Youth Risk Behavior Survey began in 1991. According to the 2013 survey results, the teen smoking rate is down to 15.7%. However, challenges remain, according to the CDC, because some surveys show an increase in hookah and e-cigarette use among teens.
The FDA approved Lymphoseek (technetium 99m tilmanocept) Injection to help doctors determine the extent of squamous cell carcinoma that has spread in the head and neck. Lymphoseek, a radioactive diagnostic imaging agent, can help identify lymph nodes closest to a primary tumor and guide sentinel lymph node biopsy. Originally approved in 2013 for use in patients with breast cancer and melanoma, Lymphoseek is marketed by Dublin, OH–based Navidea Biopharmaceuticals.
Agios Pharmaceuticals (Cambridge, MA) announced that its AG-221 received the FDA's Orphan Drug Designation, which provides additional market exclusivity upon approval, tax credits, and exemption from FDA application fees. AG-221 is an oral IDH2-mutant inhibitor being evaluated in a phase I trial in patients with advanced hematologic malignancies who carry an IDH2 mutation. In addition, data from a phase I study of the drug presented at the European Hematology Association meeting in Milan, Italy, showed objective responses in 14 of 25 evaluable patients and stable disease in five more patients; six patients achieved a complete response.
DARA Biosciences (Raleigh, NC) announced that its experimental compound KRN5500 also received Orphan Drug Designation from the FDA for the treatment of multiple myeloma. KRN5500 is a novel intravenous, nonopiod, nonnarcotic compound currently in phase II clinical development.
Myriad Genetics (Salt Lake City, UT) sued Pathway Genomics (San Diego, CA) for infringement of its patent claims on BRCA1 and BRCA2 genetic testing. The suit was spurred by Pathway's launch earlier this month of a next-generation sequencing test called BRCATrue that assesses alterations in the two genes.
The NCI renewed St. Jude Children's Research Hospital's designation as a comprehensive cancer center. Memphis, TN–based St. Jude is the only NCI-designated comprehensive cancer center devoted solely to children. A comprehensive cancer center must possess a deep and broad portfolio of laboratory, clinical, and population-based research and engage in cancer education and outreach.
The state legislature of Florida approved the expenditure of $300 million over the next 5 years to help the cancer centers at the University of Miami and the University of Florida become NCI-designated cancer centers. Tampa's Moffitt Cancer Center is currently the only NCI-designated facility in the state. Earning the designation would give the cancer centers special access to grants and clinical trials.
The U.S. Senate confirmed Sylvia Matthews Burwell as the new secretary of the Department of Health and Human Services, which includes the NIH, NCI, and the FDA. Currently the head of the Office of Management and Budget, Burwell will replace Kathleen Sebelius, who announced her resignation in April.
Cambridge, MA–based Momenta Pharmaceuticals announced that its novel oncology candidate drug, necuparanib, received orphan drug designation from the FDA for the treatment of pancreatic cancer. A heparan sulfate mimetic, necuparanib inhibits several factors implicated in tumor–host cell interactions, including VEGF and FGF2. The designation provides Momenta with tax credits, a 7-year period of marketing exclusivity following approval, and other incentives.
Eli Lilly will start sharing its clinical trial data with researchers through Clinical Study Data Request. The website, which houses data from several clinical trial sponsors, was created in support of ongoing efforts by the Pharmaceutical Research and Manufacturers of America and the European Federation of Pharmaceutical Industries and Associations to increase access to and transparency of clinical trial results for researchers.
Asymptomatic, nonpregnant adults and adolescents at high risk of hepatitis B virus (HBV) infection should be screened for it, according to a recommendation from the U.S. Preventive Services Task Force, which reviewed evidence of potential risks and benefits of screening. Published in the Annals of Internal Medicine, the recommendation is a reversal of the task force's 2004 position on HBV screening. About 15% to 25% of people with chronic HBV infection die of cirrhosis or hepatocellular carcinoma.
The Jackson Laboratory, a nonprofit biomedical research institution in Bar Harbor, ME, and Wuhan Frasergen Bioinformatics Co. announced the establishment of a cancer genomics facility in Wuhan, Hubei Province, China. The collaboration will allow Chinese researchers to investigate cancers and their response to novel drugs and immunotherapies using Jackson's severely immune-compromised mice and other innovative cancer tools.
Blind mole-rats may not have eyes, but they do possess extraordinary cancer-fighting abilities. An international team of researchers has compiled the animal's genome and reported in Nature Communications that it contains 259 defunct genes, including 22 genes involved in building the eye and processing its signals. However, the rodents have more genes involved in encoding interferon-beta1 and regulating cell death and other tumor-killing tasks than rats and mice do.
"The training of the biomedical workforce has always been an integral part of the NIH mission, and through its infrastructure of funding opportunities and other initiatives, the agency hopes to champion a culture of mentorship in the research community," wrote Sally Rockey, PhD, deputy director for extramural research at NIH, in an article in Nature Medicine on mentoring early-career scientists and the agency's mentoring programs. "It takes just one good mentor to influence the career of a new investigator; it takes a robust culture of mentorship across the research community to strengthen, sustain and diversify the entire biomedical research enterprise."
The FDA reclassified sunlamp products and UV lamps intended for use in these products from low-risk to moderate-risk devices, which will subject them to greater regulation, such as the need to obtain FDA clearance before marketing them. In addition, sunlamp products must now carry a visible black-box warning that explicitly states that they should not be used on people under age 18. Manufacturers will also need to explain the risks associated with the use of sunlamp products.
With approval from the FDA, San Diego, CA–based Halozyme Therapeutics announced that it will resume its phase II clinical trial of PEGPH20 as a first-line treatment for patients with stage IV metastatic pancreatic cancer. The company temporarily halted trial enrollment in April after a greater rate of blood clots was found in patients receiving PEGPH20; patients at high risk for blood clots will now be excluded from the trial. PEGPH20 is an investigational pegylated form of Halozyme's recombinant human hyaluronidase under development for the treatment of tumors that accumulate hyaluronan.
Private insurers and Medicare should pay for proton beam therapy for ocular melanoma, certain brain tumors, and other relatively uncommon cancers for which the treatment has proven effective, according to a "model policy" issued by the American Society for Radiation Oncology. The cost of proton beam therapy for the treatment of more common cancers, such as prostate, breast, and lung cancers, should be covered if patients are enrolled in a clinical trial, the organization said, because evidence of its effectiveness is still emerging.
Based on a pooled analysis of two clinical trials that included more than 1,800 women, Eisai (Tokyo, Japan) announced that Halaven (eribulin), a microtubule dynamics inhibitor, significantly extended overall survival in many women with breast cancer that had progressed after prior therapy. Compared with controls, women with HER2-negative disease lived 2.9 months longer (15.2 vs. 12.3) and women with triple-negative disease lived 4.7 months longer (12.9 vs. 8.2). Although women with HER2-positive breast cancer lived 1.3 months longer (13.5 vs. 12.2), the difference was not statistically significant.
About 25% of children in remission from acute lymphocytic leukemia (ALL) are missing too many doses of an essential maintenance medication that minimizes their risk of relapse, according to a study published online in Blood. The study also reports that maintenance medication adherence was lower in African American and Asian children in remission from ALL than in non-Hispanic white children, with 46% of African Americans and 28% of Asians not taking enough to prevent relapse, compared with 14% of non-Hispanic whites. Less than 90% adherence to therapy was associated with a 3.1-fold increase in the risk of relapse.
Amgen announced that the FDA approved Vectibix (panitumumab) as first-line treatment in patients with wild-type KRAS metastatic colorectal cancer (mCRC), to be used in combination with FOLFOX, an oxaliplatin-based chemotherapy regimen. The FDA also approved the therascreen KRAS test developed by QIAGEN as a companion diagnostic for Vectibix. Vectibix was first approved in 2006 as a monotherapy for the treatment of patients with EGFR-expressing mCRC after prior chemotherapy.
The Ohio State University Comprehensive Cancer Center in Columbus and the Moffitt Cancer Center in Tampa, FL, have launched the Oncology Research Information Exchange Network (ORIEN), which includes deidentified clinical data and tissue samples from more than 100,000 patients, to accelerate the development of targeted treatments and to more quickly match eligible patients to clinical trials. ORIEN will point physicians to the best therapeutic options for each patient's biologic and epidemiologic profile, increasing the likelihood that treatment will be effective. Patients will be followed through their lifetime, the institutions said, improving care for future generations.
BioAlliance Pharma (Paris, France) and Topotarget (Copenhagen, Denmark) announced that their combined entity will be named Onxeo and will specialize in developing medicines for "orphan oncology conditions." The merger, announced in April, will be finalized this summer.
Asymptomatic, nonpregnant adolescents and adults who are at high risk of becoming infected with the hepatitis B virus should been screened for it, according to a final recommendation issued by the U.S. Preventive Services Task Force (USPSTF). The recommendation does not apply to people who have been vaccinated against the virus, which can cause liver cancer. The USPSTF didn't have enough evidence to determine appropriate screening intervals.
The American Society of Clinical Oncology updated its clinical practice guideline on the use of adjuvant endocrine therapy for women with hormone receptor–positive breast cancer, noting that data from recent studies suggest that women who take tamoxifen for 10 years live longer and have lower risks of breast cancer recurrence and contralateral breast cancer, compared with women who take the drug for 5 years. The guideline is available at www.asco.org/guidelines/breast-cancer.
In a study published in JAMA Surgery, researchers found that nearly 70% of women who undergo prophylactic contralateral mastectomy have no clinically significant risk factors for developing cancer in the unaffected breast. About 80% of those women said they opted for surgery to prevent disease recurrence even though the procedure has not been shown to reduce the risk of recurrence.
The FDA granted Breakthrough Therapy designation to Clovis Oncology's CO-1686 and to Bristol-Myers Squibb and AbbVie's elotuzumab. CO-1686 is being tested as a monotherapy for the treatment of second-line EGFR-mutant non–small cell lung cancer in patients with the T790M mutation. Elotuzumab is an investigational, humanized monoclonal antibody for use with lenalidomide and dexamethasone for the treatment of multiple myeloma in patients who have received one or more prior therapies.
According to projections from the Pancreatic Cancer Action Network in Manhattan Beach, CA, pancreatic and liver cancers will become the second and third leading causes of cancer-related death in the United States by 2030, surpassing breast, prostate, and colorectal cancers. Lung cancer will remain the top killer. Breast, prostate, and lung cancers will remain the most common cancer diagnoses.
Livatag (doxorubicin Transdrug) received Fast Track designation from the FDA for second-line treatment of hepatocellular carcinoma following therapy with sorafenib (Nexavar; Bayer and Onyx Pharmaceuticals). Under development by BioAlliance Pharma in Paris, France, Livatag is a chemotherapy drug using a nanoparticle formulation.
In an effort to combat lung cancer and other serious diseases, The University of Texas MD Anderson Cancer Center will adopt a tobacco-free hiring policy beginning January 1, 2015. All job seekers will be screened for tobacco use and those who test positive will not be eligible for immediate employment. Applicants who remain interested in employment will be given tobacco-cessation materials and instructions for obtaining assistance in quitting, and they can reapply after 6 months.
Writing in the Journal of Clinical Oncology, three physicians at The University of Texas Health Science Center at San Antonio say that cancer researchers need to recruit more Hispanic patients for clinical trials. "How else will we know how best to treat our patients, and how else are we going to reduce health disparities in this population?" says Ian Thompson, MD, director of the institution's Cancer Therapy & Research Center. The population of South Texas is 68% Hispanic; however, fewer than 4% of participants in clinical trials, on average, are Hispanic.
Based on data from the NIH Human Microbiome Project, 69% of healthy Americans are infected with at least one strain of human papillomavirus (HPV), researchers reported at the American Society for Microbiology's annual meeting in Boston, MA. However, only four of the 103 men and women whose tissue was used in the analysis had either of the two HPV types known to cause most cases of cervical cancer—HPV 16 and 18. In all, the researchers found 109 of the 148 known types of HPV.
New York, NY's Memorial Sloan Kettering Cancer Center received a gift of $100 million from the Marie-Josée and Henry R. Kravis Foundation to support the institution's Center for Molecular Oncology, which will be named in their honor. The center aims to profile 10,000 patient tumors in its first year, using next-generation sequencing and other technologies. Molecular information for each tumor will then be correlated with clinical outcomes to better understand the significance of genetic alterations in tumors and treat cancers more precisely.
Cervical cancer rates in the United States are higher than previously believed, according to a study published in Cancer. Previous research found about 12 cases of cervical cancer per 100,000 women, with incidence peaking between ages 40 and 44, but those estimates included women who had had their cervix removed. When these women are excluded from the analysis, the rate increased to 18.6 cases per 100,000 women, the new study found, with incidence peaking between ages 65 and 69; current guidelines say screening can stop at age 65 for many women.
According to a survey of more than 1,000 women by the American Lung Association, women are largely unaware of their risk of lung cancer. Despite being the leading cause of cancer mortality among women, lung cancer was cited by just 1% of women as their primary cancer concern, and 78% of women didn't know that the disease has killed more women than breast cancer since 1987.
The American Society of Clinical Oncology (ASCO) released resources to help physicians manage the complications of obesity in cancer and facilitate communication about the importance of weight management and healthy lifestyle choices following a cancer diagnosis. Obesity is not only a risk factor for multiple types of cancer, but also a factor in poor treatment outcomes.
Regardless of socioeconomic status, black women are nearly twice as likely to be diagnosed with triple-negative breast cancer as white women, according to an analysis published in Breast Cancer Research and Treatment. The researchers also found that Asian/Pacific Islander women had higher odds of presenting with HER2-overexpressing breast cancer than white women.
Waltham, MA–based Tesaro announced that rolapitant, a drug it is developing to ease chemotherapy-related nausea and vomiting, met its primary and secondary endpoints in a phase III trial. The company said it will release data from the trial at the ASCO annual meeting, which begins on May 30, and apply for FDA approval in mid-2014.
The oral combination anticancer drug TAS-102 (trifluridine and tipiracil hydrochloride) demonstrated improvement in overall survival in patients with refractory metastatic colorectal cancer whose disease had progressed despite receiving at least two prior chemotherapy regimens, meeting its primary endpoint, according to Taiho Oncology (Princeton, NJ). The international, phase III RECOURSE trial included 800 patients who were randomly assigned to receive TAS-102 or a placebo. Detailed results will be presented at the European Society of Medical Oncology's 16th World Congress on Gastrointestinal Cancer in Barcelona, Spain, in June.
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins in Baltimore, MD, received a gift of $65 million to fund construction of a new patient care building that will be named for the late Albert P. "Skip" Viragh Jr., a mutual fund investment leader and philanthropist. A patient at Hopkins, Viragh died of pancreatic cancer in 2003.
The American Society for Radiation Oncology urged the Centers for Medicare and Medicaid Services (CMS) to support a U.S. Preventive Services Task Force (USPSTF) proposal that recommends annual lung cancer screening with low-dose CT (LDCT) in adults ages 55 to 80 who have a 30 pack-year smoking history and who currently smoke or have quit smoking within the past 15 years. Last week, a CMS advisory panel said Medicare shouldn't cover the screenings because there isn't enough evidence that the benefit of annual LDCT screening outweighs the harms. Medicare is not required to follow USPSTF recommendations, although private insurers are.
Customers at more than 8,000 Walgreens stores contributed $15 million during 2012 and 2013 for Susan G. Komen, the world's largest breast cancer organization. Last year, the Walgreens program allowed Komen to fund more than 15,000 mammograms for uninsured or underinsured women, and supported education and patient navigation services for another 220,000 people. The money will also help fund the Susan G. Komen Helpline for the next 3 years, the charity announced.
The American Society of Clinical Oncology issued two clinical practice guidelines for treating women with advanced, HER2+ breast cancer. The first guideline lists appropriate systemic therapies for women newly diagnosed with advanced disease and those whose early-stage disease progressed to advanced cancer. The second guideline provides recommendations for treating brain metastases in women with the disease.
Radiologists in New Jersey must now tell women who have a mammogram that they may have dense breasts and should discuss the issue with their doctors. New Jersey is the 15th state to pass such a law. Dense breasts have more connective tissue and less fat, making it harder to spot tumors, because connective tissue and tumors both appear white on mammograms.
An in-depth analysis of data from one of the Women's Health Initiative (WHI) Postmenopausal Hormone Therapy Trials has found that the investment in the WHI by the NIH resulted in a return of $140 for each dollar spent on the trial. In addition, they found that guidance provided by the trial's results led to 76,000 fewer cases of cardiovascular disease; 126,000 fewer cases of breast cancer; 145,000 more quality-adjusted life years; and savings of $35.2 billion on direct medical expenses.
The FDA granted "Priority Review" designation to Merck's investigational anti–PD-1 antibody MK-3475 for the treatment of advanced melanoma. A decision is anticipated by October 28.
Teva Pharmaceuticals (Jerusalem, Israel) announced that the FDA approved injectable Synribo (omacetaxine mepesuccinate) for home use. Physicians can now allow adults with chronic myeloid leukemia who are no longer responding to, or cannot tolerate, two or more tyrosine kinase inhibitors to administer the therapy at home.
A study published in JAMA Dermatology found that the risk of developing cancer from UV lamps used to dry and cure polish at nail salons is relatively small because exposure to the lamps after a manicure is brief and multiple visits would be required to potentially cause DNA damage. Higher-wattage UV lamps have been associated with greater emission of UV-A radiation, prompting concern about cancer.
Johnson & Johnson suspended sales of its laparoscopic power morcellators, devices used to remove uterine fibroids, until their role in treatment is more clearly determined. Last month, the FDA recommended against using the devices amid concerns that they may spread undetected cancer.
Novartis announced that the FDA granted accelerated approval to Zykadia (ceritinib) for the treatment of patients with metastatic, ALK-positive non–small cell lung cancer who have progressed on or are intolerant to crizotinib (Xalkori; Pfizer), the only other approved ALK inhibitor. Among 163 previously treated patients, Zykadia achieved an overall response rate of 54.6% and a median duration of response of 7.4 months.
The FDA approved the cobas HPV DNA test for primary cervical cancer screening in women ages 25 and older. Manufactured by Roche Molecular Systems, the test can also provide information about a patient's risk for developing cervical cancer in the future. The agency first approved the test in 2011 for use in conjunction with or as a follow-up to Pap testing.
In a phase III trial, patients with metastatic pancreatic cancer who received the combination of Merrimack Pharmaceuticals' experimental drug MM-398, 5-fluorouracil (5-FU), and leucovorin achieved an overall survival of 6.1 months compared with 4.2 months in patients who received 5-FU and leucovorin alone. Patients who took MM-398 as monotherapy had a median overall survival of 4.9 months, which was not statistically significant. MM-398 is a nanoliposomal encapsulation of the chemotherapeutic irinotecan, which, in its activated form, inhibits topoisomerase I, an enzyme involved in DNA transcription and replication.
GW Pharmaceuticals (London, UK) announced that the FDA granted Fast Track designation to Sativex for the treatment of pain in patients with advanced cancer who experience insufficient analgesia from opioid therapy. Composed primarily of two cannabinoids—cannabidiol and delta 9 tetrahydrocannabinol—Sativex is under evaluation in a phase III trial. It is administered as a metered-dose oromucosal spray.
Four Canadian government research funding agencies will spend $6.7 million to develop powerful new computing tools so that researchers can analyze genetic data from thousands of cancers. At the heart of the project is a new cloud computing facility, the Cancer Genome Collaboratory, capable of processing genetic profiles collected by Toronto's International Cancer Genome Consortium from cancers in some 25,000 patients. The facility will open to the broader research community in 2016.
At a time when "the scientific possibilities have never been more promising," NIH Director Francis Collins, MD, PhD, told members of the U.S. Senate Appropriations Committee that the country is losing its position as the world's leader in science and innovation due to flat federal research funding, irregular budget processes, sequestration, and inflation. "What we desperately need is a new bipartisan plan to secure a steady funding trajectory for biomedical research and ensure long-term stability for NIH's mission."
As part of its implementation of the 2009 Family Smoking Prevention and Tobacco Control Act, the FDA issued a proposed rule that would extend its authority to regulate additional tobacco products including electronic cigarettes, cigars, pipe tobacco, nicotine gels, waterpipe (or hookah) tobacco, and dissolvables. The FDA currently regulates cigarettes, cigarette tobacco, roll-your-own tobacco, and smokeless tobacco.
Novartis announced that it will acquire GlaxoSmithKline's (GSK) oncology products for as much as $16.1 billion and send its vaccines business (excluding flu vaccines) to GSK for $7.1 billion plus royalties. GSK oncology drugs include the BRAF inhibitor Tafinlar (dabrafenib) and the MEK inhibitor Mekinist (trametinib), both for metastatic melanoma, and the VEGFR inhibitor Votrient (pazopanib) for renal cell carcinoma. The two companies also agreed to combine their consumer divisions to create a world-leading consumer healthcare business.
European biotechs BioAlliance Pharma (Paris, France) and Topotarget (Copenhagen, Denmark) have agreed to merge and combine their pipelines of late-stage oncology drugs. Topotarget's leading drug is belinostat, an HDAC inhibitor for use in certain lymphomas, currently under FDA review. BioAlliance's oncology pipeline includes Livatag, a treatment for primary liver cancer that is currently in phase III trials.
Indianapolis, IN–based Eli Lilly received FDA approval to market Cyramza (ramucirumab) as a single-agent treatment for patients with advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma with disease progression. Approval was based on results of REGARD, a multicenter, randomized, placebo-controlled, double-blind trial that showed the addition of Cyramza to best supportive care increased median overall survival by 37% (5.2 months vs. 3.8 months) and increased median progression-free survival by 62% (2.1 months vs. 1.3 months).
The FDA proposed a new program to provide earlier access to high-risk medical devices intended to treat or diagnose patients with serious conditions whose medical needs are unmet by current technology. The program features earlier and more interactive engagement with FDA staff and a collaboratively developed plan for collecting scientific and clinical data to support approval. While other programs have focused on reducing the time needed for premarket review, this program seeks to reduce the time associated with product development.
Saying it poses a risk of spreading an undetected cancer, the FDA issued a safety notice discouraging the use of laparoscopic power morcellation to remove the uterus or uterine fibroids. The technique cuts up uterine tissue so it can be removed through a small abdominal incision.
The FDA converted the accelerated approval of GSK's ofatumumab (Arzerra) to regular approval based on the results of a multicenter, randomized, open-label trial comparing ofatumumab plus chlorambucil to single-agent chlorambucil in 447 patients with chronic lymphocytic leukemia who weren't candidates for fludarabine-based therapy. Progression-free survival was 22.4 months for patients receiving the combination compared with 13.1 months for patients receiving chlorambucil alone. As part of the accelerated approval, which was granted in 2009, GSK was required to conduct further studies of ofatumumab.
The NIH announced a change in policy that will allow researchers, after an unsuccessful resubmission application, to submit the same idea as a new application. "The resubmission of an idea as new means the application will be considered without an association to a previous submission," blogged Sally Rockey, PhD, deputy director for extramural research at NIH. "The applicant will not provide an introduction to spell out how the application has changed or respond to previous reviews, and reviewers will be instructed to review it as a new idea, even if they have seen it in prior cycles."
Noting that manufacturers rely on marketing practices that appeal to youth, a Congressional report on e-cigarettes released on April 14 calls upon the FDA to "act quickly" to regulate the devices. The report, written by the staff of 11 senators and representatives, also says that manufacturers doubled their spending to market e-cigarettes between 2012 and 2013. Mitchell Zeller, JD, director of the FDA's Center for Tobacco Products, said earlier this month that a regulatory document is currently under review at the Office of Management and Budget and would be released soon, although he could not give a date.
Cambridge, MA–based Foundation Medicine disclosed that it filed a request with the Securities and Exchange Commission to withdraw its proposed public offering of $150 million in common stock. The cancer genetics diagnostics company submitted paperwork to the federal agency for the public offering in March.
British researchers reported a steep decline in the number of young women infected with the two strains of human papillomavirus (HPV) most likely to cause cervical cancer occurred in England after the launch of an HPV vaccination program there in 2008. Based on test results from more than 4,000 women between 2010 and 2012, one in 15 sexually active females ages 16 to 18 was infected with either HPV16, HPV18, or both, down from one in five before the start of the program. The findings were presented at the annual meeting of the Society for General Microbiology in Liverpool.
According to a prospective, single-arm study, 28% of 157 patients pursuing active surveillance for low-risk prostate cancer experienced disease progression over 13 years of follow-up and required definitive treatment, leading to a cancer-specific survival rate of 99%. However, about 27% of the men in the trial did not show up for checkups or reply to follow-up letters, casting some doubt on the safety of active surveillance. The findings were reported at the European Association of Urology (EAU) 29th Annual Congress in Stockholm, Sweden
Also at the EAU meeting, Monique Roobol, PhD, an associate professor of urology at the University of Erasmus Medical Center in Rotterdam, the Netherlands, said that PSA screening "is here to stay" in spite of the ongoing debate about its risks and potential benefits. Physicians, she said, should abandon a one-size-fits-all approach to screening, prepare to discuss the pros and cons of screening with patients, and account for factors other than PSA when assessing an individual's risk, because population-based estimates are difficult to translate to a particular patient.
Warning that the biomedical research enterprise in the United States is "under great stress and at risk for incipient decline," four prominent researchers, including Harold Varmus, director of the NCI, call for multiple systemic changes in an opinion piece in the Proceedings of the National Academy of Sciences. They write that budgets for scientific funding agencies need to be more predictable, that the number of PhD students should be brought in line with the number of future opportunities, and that grant reviewers should "put a higher priority on the quality, novelty, and long-term objectives of the project than on technical details." Such changes, they say, "need to be made in a comprehensive fashion, not piecemeal… need to be made with extreme care… and need to begin immediately."
The American Society of Clinical Oncology released three clinical practice guidelines to address fatigue, peripheral neuropathy, and anxiety and depression, health issues frequently reported by cancer survivors. The recommendations, part of a planned series of 18 such guidelines, are available at www.asco.org/guidelines/survivorship.
The National Cancer Institute (NCI) announced that formal awards for the National Clinical Trials Network would be made in the coming weeks and that funding levels for clinical trials in fiscal year (FY) 2014 would be unchanged from FY2013. However, the number of trials that can be funded will be lower than in the past. "The operational concessions are required to provide higher reimbursement rates per patient to the academic sites that are developing and performing trials," according to the NCI statement.
The University of Texas MD Anderson Cancer Center in Houston announced that it formed a research alliance with pharmaceutical giant GlaxoSmithKline to more rapidly develop cancer immunotherapies. Ferran Prat, PhD, JD, vice president of Strategic Industry Ventures, said that this is MD Anderson's fourth and final major collaboration with a large company on immunotherapy. The other three companies are Pfizer, Johnson & Johnson, and MedImmune.
The agent IMCgp100, a new type of immunotherapy, was well tolerated and showed efficacy in some patients with advanced melanoma in a phase I clinical trial, according to data reported on April 8 at the American Association for Cancer Research (AACR) Annual Meeting in San Diego, CA. IMCgp100 attaches to cancer cells, activates any adjacent T cells to kill the cancer cells, and recruits other immune cells to help clear the disease, explained Mark Middleton, MD, PhD, professor of experimental cancer medicine at the University of Oxford in the UK. "The ability of IMCgp100 to target one of a largely unexplored class of molecular targets, HLA-peptides, opens the door to the treatment of many forms of cancer for which no antibody-applicable target has yet been identified," Middleton said.
Speaking at a panel discussion at the AACR meeting, Sharon Plon, MD, PhD, called upon cancer organizations to put together their own guidelines for handling the reporting of incidental findings to patients who undergo tumor genome analysis. A professor of molecular and human genetics at Baylor College of Medicine in Houston, TX, Plon noted that no oncologists were part of the group that developed the American College of Medical Genetics and Genomics' guidelines, which were issued last year and updated last month. Those guidelines, the only ones in existence in the United States, list 56 genes, including 23 cancer susceptibility genes, for which variants should be reported to patients who undergo clinical sequencing, even if they uncover a medical condition not related to the original reason for the test.
According to results of a first-in-human phase I trial presented on April 7 at the AACR meeting, the investigational antibody–drug conjugate DEDN6526A (Genentech) was safe, tolerable, and showed hints of activity against cutaneous, mucosal, and ocular melanoma. Clinical benefit was observed in 12 of the 19 patients who received 1.8 mg/kg or more of the drug; four of these patients, two with cutaneous melanoma and two with mucosal melanoma, had confirmed partial responses. The antibody portion of the agent recognizes the protein endothelin B receptor, and the toxic chemotherapy attached to it is monomethyl auristatin.
"Senescence is the underappreciated stepchild of cancer biology," said Norman Sharpless, MD, director of the Lineberger Comprehensive Cancer Center in Chapel Hill, NC, who chaired a session on senescence, cancer, and aging at the AACR meeting. Cellular senescence seems to be a critical defense against cancer, as well as a determinant of how humans age. Expression of p16 INK4a increases markedly with aging and can serve as a biomarker of physiologic aging, he said.
In a special symposium on data reproducibility in cancer research, Ferric Fang, MD, a professor of laboratory medicine and microbiology at the University of Washington School of Medicine in Seattle, said that cancer research papers are overrepresented among those listed as retracted in PubMed. He noted that the costs of research misconduct are in the hundreds of millions of dollars.
"Overweight and obesity are contributing just about as many excess cancers now in the United States as is current smoking," commented Walter Willett, MD, MPH, DrPH, of the Harvard School of Public Health in Boston, MA, during a presentation on diet and cancer at the opening plenary session of the AACR meeting on April 6. Yet because fewer than 20% of Americans currently smoke while more than two thirds are obese, "on an individual basis, it's still worse to smoke than to be overweight or obese," he said.
Also at the meeting, researchers presented phase I trial data showing that the experimental anti–PD-1 monoclonal antibody MK-3475 improves progression-free survival in advanced melanoma and in non–small cell lung cancer. "There are multiple assays in development for PD-1 and PD-L1… Do they correlate? Are they consistent?" noted Mario Sznol, MD, of Yale Cancer Center in New Haven, CT, who discussed the findings. "And, there's heterogeneity within and between metastases in a single patient, so there's a real chance for false negatives, and that's a real problem because the alternative treatments for these patients are very poor."
The AACR formally inducted its 2014 class of 38 elected fellows of the AACR Academy on April 4, just before the start of its annual meeting, held in San Diego, CA, April 5–9. The AACR Academy was created in 2013 to recognize and honor distinguished scientists whose major scientific contributions have propelled significant innovation and progress against cancer. The fellows are elected through a rigorous peer-review process.
The American College of Medical Genetics and Genomics released guidelines saying that a patient should be allowed to opt out of recommended testing for genetic mutations that might indicate an increased risk of certain medical conditions, including some cancers. The new recommendation replaces an earlier one that called for doctors to automatically screen for genetic mutations linked to 24 medical conditions—even if the condition has no bearing on the original reason for the test. All 24 medical conditions have treatment options and can possibly be prevented, but ethicists argue that patients should have the right to decide whether they want this additional testing.
GlaxoSmithKline announced that its cancer vaccine MAGE-A3 failed to meet two primary endpoints in a phase III non–small cell lung cancer (NSCLC) trial. Last September, the company announced that the investigational immunotherapy failed to outperform a placebo in a phase III melanoma trial. Researchers will continue to analyze results, however, to see if subpopulations of cancer patients might benefit from MAGE-A3.
The U.S. Food and Drug Administration (FDA) determined that Curis, Inc., can continue a phase I trial of CUDC-427, a small-molecule Smac mimetic designed to block proteins that inhibit apoptosis, in solid tumors and lymphoma. The FDA put a partial hold on the trial in November after a patient died of liver failure a month after discontinuing the drug. The partial hold meant that the Lexington, MA–based company could not enroll new patients in the trial.
Among 114 patients with ALK+ NSCLC in a phase I single-arm study who took at least 400 mg of ceritinib (LDK378; Novartis) daily—including patients whose disease had progressed on the ALK inhibitor crizotinib—the overall response rated was 58%, according to data published in The New England Journal of Medicine. Among the responders, the median duration of response was 8.2 months. Researchers noted that developing new therapies for NSCLC is critical because most patients relapse within a year of starting therapy with crizotinib (Xalkori; Pfizer) and have few other treatment options.
Biogen Idec has agreed to provide $5.25 million over the next 3 years to support basic biomedical research projects led by investigators at the Whitehead Institute for Biomedical Research. The funded projects, which will be determined by a committee of scientists from both of the Cambridge, MA, organizations, will focus primarily on immunology, neurology, developmental biology, genetics, and genomics. "This agreement will enable the launch of research projects at a level that might not attract funding from more traditional sources," said David Page, director of the Whitehead Institute.
Based on a phase II study presented at the Society of Gynecologic Oncology Annual Meeting in Tampa, FL, gynecologic cancer cells with a BRCA mutation appear to be sensitive to monotherapy with AbbVie's PARP inhibitor veliparib. Overall, 13 of the 50 patients in the trial experienced tumor shrinkage, and 24 experienced stable disease for 4 months. Research had previously shown that veliparib is effective in combination with chemotherapy, but data about the effectiveness of the drug as a single agent have been lacking.
Foundation Medicine has filed with the U.S. Securities and Exchange Commission to sell up to $150 million in common stock in a public offering. The Cambridge, MA-based firm uses next-generation sequencing to provide physicians with molecular information about a patient's cancer that could help guide clinical care.
South San Francisco, CA–based Exelixis announced that the European Commission approved Cometriq (cabozantinib) for the treatment of adults in the European Union withprogressive, unresectable locally advanced or metastatic medullary thyroid carcinoma. The drug, which inhibits the receptor tyrosine kinases RET, MET, and VEGF, was approved by the FDA for this indication in November 2012.
Novant Health Presbyterian Medical Center in Charlotte, NC, received a $10 million gift to fund the construction of a new building that will house outpatient services for patients with cancer and heart disease. The family of the late John Belk, a former mayor of the city and an executive at the Belk department store chain, made the donation.
According to a multicenter trial published in The New England Journal of Medicine, Cologuard, a noninvasive, stool-based DNA test to screen for colorectal cancer, had detection rates similar to those reported for colonoscopy and better than fecal immunochemical testing (FIT). The sensitivity of Cologuard (Exact Sciences; Madison, WI) for cancer was 92% overall, compared with 74% for FIT; its sensitivity for detecting advanced precancerous lesions was 42%, compared with 24% for FIT. Because the samples can be collected and mailed from home, researchers hope that more patients will opt for screening if the test is approved by the U.S. Food and Drug Administration (FDA).
Pfizer announced that its ALK inhibitor Xalkori (crizotinib) significantly prolonged survival in a phase III trial of previously untreated patients with ALK-positive non-squamous non–small cell lung cancer compared with standard chemotherapy. Xalkori received FDA approval in 2011 for use in ALK-positive lung cancer patients as a second-line therapy. The new data, which will be presented at an upcoming scientific meeting, demonstrate its effectiveness as a first-line treatment.
E-cigarettes are promoted as a smoking-cessation tool, but in a new study, researchers found that the use of the devices by current smokers is not associated with greater rates of quitting smoking or reduced cigarette consumption after a year. Although only 88 of the 949 smokers in the study used them when the study started, "regulations should prohibit advertising claiming or suggesting that e-cigarettes are effective smoking-cessation devices until claims are supported by scientific evidence," the researchers wrote in JAMA Internal Medicine.
Attorneys general from 28 states and territories sent letters to five of America's largest retail chains, encouraging them to stop selling tobacco products in stores that have a pharmacy. Kroger, Rite Aid, Safeway, Walgreens, and Walmart were asked to consider following the example set by CVS Caremark, which announced last month that it would stop selling tobacco in all of its stores by October 1.
In a deal worth up to $350 million, Bristol-Myers Squibb is pairing up with Five Prime Therapeutics (South San Francisco, CA) to develop new immune-oncology therapies to target two immune checkpoint pathways. The company did not specify the pathways. Bristol-Myers, which is developing the immunotherapeutic nivolumab, will hold the rights to any products developed as a result of the collaboration.
In a retrospective analysis of a phase III trial of Amgen's cancer vaccine talimogene laherparepvec, or T-Vec, researchers found that about two thirds of melanomas injected with the vaccine shrank by at least half. In addition, 15% of visceral tumors—those in organs such as the liver and lungs—and one third of nonvisceral tumors that were not injected also shrank by at least half, suggesting that the vaccine can stimulate a systemic antitumor response. Researchers are now assessing whether the vaccine will improve overall survival.
The earliest known case of metastatic cancer was discovered in a 3,000-year-old skeleton—likely belonging to a man who was 25 to 35 years old when he died—unearthed at an archeological site in northern Sudan. Although they could not pinpoint the exact disease, researchers said that lesions on the bones, which they analyzed with radiography and a scanning electron microscope, were likely caused by a soft-tissue cancer.
Findings from a report in CA: A Cancer Journal for Clinicians show that the rate at which people are diagnosed with colorectal cancer has declined by 30% in the past decade among people age 50 and older. The researchers attribute the decreased incidence to more people getting screened for the disease as recommended.
The UK has unveiled its Early Access to Medicines Scheme, a program designed to help dying patients access unapproved but potentially life-saving experimental medications. Next month, the UK's Medicines and Regulatory Healthcare Products Agency will begin accepting applications from drugmakers who want to participate. To qualify, a drug must first receive a "promising innovative medicine" designation based on early clinical data; additional data must demonstrate sufficient quality, safety, and efficacy if the drug is to be given to patients before it is officially licensed.
Florida Governor Rick Scott is touting his budget proposal to spend $80 million to help cancer centers in the state qualify as National Cancer Institute (NCI)–designated facilities. "Florida has the second-highest number of cancer cases in the country, but only one NCI-designated cancer center," University of Miami President Donna Shalala told the Miami Herald. She wants the school's Sylvester cancer center to become the second, behind Tampa's Moffit Cancer Center, a process that could take several years.
The U.S. District Court for the District of Utah denied a request from Salt Lake City's Myriad Genetics and other patent holders for a preliminary injunction against Ambry Genetics to prevent it from selling tests to assess BRCA1 and BRCA2 mutations. Myriad filed suit against Ambry, of Aliso Viejo, CA, in July 2013, alleging that Ambry's tests infringed on patent claims that were not invalidated when the U.S. Supreme Court ruled in June that genes cannot be patented because they are products of nature. The new ruling means that Ambry can continue to offer its BRCA tests pending the lawsuit's outcome.
With $70 million in investor funding, Human Longevity Inc., a genomics and cell therapy–based diagnostic and therapeutic company focused on extending the length and quality of the human life span, was founded by J. Craig Venter, PhD, Robert Hariri, MD, PhD, and Peter Diamandis, MD, in San Diego, CA. The company aims to build the world's largest human sequencing operation "to compile the most comprehensive and complete human genotype, microbiome, and phenotype database available to tackle diseases associated with aging-related human biological decline." The University of California, San Diego, Moores Cancer Center will work with the company to conduct genomic sequencing of patients' tumors.
The American Society of Clinical Oncology issued a comprehensive report on the cancer-related challenges facing the U.S. medical system. The State of Cancer Care in America: 2014 estimates that, due to an aging population, the number of cancer cases will increase by as much as 42% by 2025 while the number of oncologists will likely grow by just 28%, creating a shortage of nearly 1,500 doctors. In addition, the cost of cancer care, the report says, is likely to increase by 40% between 2010 and 2020.
German regulators granted Northwest Biotherapeutics of Bethesda, MD, special permission to sell its experimental brain cancer immunotherapy, DCVax-L, even though late-stage trials have not yet been completed. Regulators said the drug could be used after patients receive standard care, which includes surgery, radiation, and chemotherapy. In small, early-phase trials, patients with glioblastoma multiforme who received standard care and DCVax-L lived more than twice as long as patients who received standard care only.
Bayer HealthCare announced that a phase III trial evaluating its drug Nexavar (sorafenib) as an adjuvant treatment for patients with hepatocellular carcinoma did not improve recurrence-free survival, the primary endpoint. The drug is already approved for advanced kidney and liver cancers. Data from the study will be presented at an upcoming scientific meeting, according to the company.
Lexington, MA–based Synta Pharmaceuticals announced that its Hsp90 inhibitor ganetespib has been selected for study as part of I-SPY 2, a standing phase II randomized, controlled, multicenter trial for women with newly diagnosed, locally advanced breast cancer designed to test whether adding investigational drugs to standard chemotherapy is better than standard chemotherapy alone prior to surgery. The trial uses biomarkers from patients' tumors to match them with experimental treatments.
Already approved to treat myelofibrosis, Jakafi (ruxolitinib) met its primary endpoint in a phase III trial that included 222 patients with polycythemia vera, a rare blood cancer. Incyte (Wilmington, DE), which holds domestic rights to the JAK1/JAK2 inhibitor, said it expects data to be presented at an upcoming scientific meeting.
Cerulean Pharma is seeking $75 million through an initial public offering to further develop CRLX101, which failed to meet its primary endpoint of overall survival in a phase IIb study in patients with non–small cell lung cancer last year. The Cambridge, MA, company wants to further test the treatment, a nanopharmaceutical that links the cancer-killing agent camptothecin to proprietary polymers that target tumors, in renal cell carcinoma, ovarian cancer, and rectal cancer.
The U.S. Senate passed legislation to repeal taxpayer subsidies for political conventions and divert the savings, estimated to be $126 million over 10 years, to research on pediatric cancer and other childhood diseases. Majority Leader Harry Reid noted that the measure was largely symbolic and that it wouldn't directly fund research, which occurs as part of appropriations bills. "It's extremely important that we understand that the NIH is billions of dollars short of being able to maintain the place they've had in the past," he said.
President Obama released his budget proposal for fiscal year (FY) 2015, which recommends $30.2 billion for the NIH, a $300 million increase over the current funding level of $29.9 billion; the National Cancer Institute would receive a $2 million increase, bringing its budget to $4.931 billion. Even with the proposed increases, however, these amounts fall short of FY 2012 budgets.
The European Parliament approved new rules to ban advertising for e-cigarettes beginning in mid-2016. E-cigarettes would also be required to carry graphic health warnings on their packaging, limit nicotine content, and be childproof. The legislation still needs the approval of all 28 members of the European Union, which experts say will likely happen in the next few weeks.
Blaming worsening air pollution, the Beijing office of China's Cancer Prevention and Control Center reported a steep rise in lung cancer cases in the city. Between 2002 and 2010, for every 100,000 men living in the city, the number of cases of lung cancer rose from 49 to 75; for every 100,000 women, the number of cases jumped from 30 to 46. According to the World Health Organization's World Cancer Report 2014, one third of global deaths from lung cancer occur in China, which accounts for about one fifth of the world's population.
San Diego, CA–based MEI Pharma announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation to Pracinostat, its investigational drug for the treatment of acute myeloid leukemia. The designation earns the company certain tax credits, fee exemptions, and 7 years of marketing exclusivity for the drug upon its approval. A histone deacetylase (HDAC) inhibitor, Pracinostat has been tested in phase I and phase II clinical trials of advanced hematologic diseases and solid tumors in adults and children.
The FDA also granted orphan drug designation to the proteasome inhibitor marizomib for the treatment of multiple myeloma, according to its developer, Triphase Accelerator Corporation of San Diego and Toronto, Canada. An intravenous formulation has been evaluated in more than 230 patients in four phase I and phase II studies, either as a single agent or in combination with the steroid dexamethasone or an HDAC inhibitor.
Due to a lack of clinically meaningful efficacy, an independent data-monitoring committee recommended that Roche halt a phase III study of its onartuzumab (MetMab) in patients with previously treated, advanced non–small cell lung cancer whose tumors were identified as MET-positive. The 499 patients in the trial received all received erlotinib (Tarceva; Genentech/Astellas) plus either onartuzumab or a placebo. The primary endpoint was overall survival.
Children's Hospital of Eastern Ontario (Ottawa, Canada), in conjunction with the University of Toronto's Department of Computer Science, announced the launch of PhenomeCentral, a database for genetic and phenotypic information on rare diseases. Physicians from around the world can upload de-identified information on their patients at PhenomeCentral, and the database will find others in the system with a similar genotype and phenotype, "enabling faster diagnoses and simpler identification of the genetic cause of rare diseases," says Michael Brudno, an associate professor at the University who also holds an appointment at The Hospital for Sick Children in Toronto. Several Canadian organizations funded PhenomeCentral.
In guidelines published in the Annals of Internal Medicine, the United States Preventive Services Task Force recommends against using vitamin E and ß-carotene supplements to reduce the risk of cancer and cardiovascular disease because evidence suggests they offer no net benefit. In addition, the group found adequate evidence that ß-carotene supplements increase the risk for lung cancer among people at increased risk of the disease, such as smokers.
For the first time, the U.S. Food and Drug Administration (FDA) halted the distribution and sale of certain tobacco products currently on the market, the authority for which was granted to the agency under the 2009 Family Smoking Prevention and Tobacco Control Act. Manufactured by Jash International, the four products—Sutra Bidis Red, Sutra Bidis Menthol, Sutra Bidis Red Cone, and Sutra Bidis Menthol Cone—were found to be not substantially equivalent to tobacco products commercially marketed as of February 15, 2007, and the company did not meet FDA requirements to continue selling them. Bidis are thin, hand-rolled cigarettes filled with tobacco and wrapped in leaves from a tendu tree that are tied with string.
Eli Lilly announced that its angiogenesis inhibitor ramucirumab showed a statistically significant improvement in overall survival in a global phase III trial comparing ramucirumab plus docetaxel to a placebo plus docetaxel in more than 1,200 patients with non–small cell lung cancer. The patients who received ramucirumab also experienced a statistically significant improvement in progression-free survival. The company said that full data will be presented at an upcoming scientific meeting.
Alabama's state legislature approved a bill that would restrict the use of tanning beds at salons by anyone under age 18 by requiring a doctor's prescription, in-person parental consent, or a note from a parent, depending on the minor's age. The governor is expected to sign the bill. The legislatures in Washington State and Colorado are considering proposals that would ban the use of commercial tanning beds by anyone under age 18.
The Van Andel Institute announced the establishment of a global epigenetics research hub to look for new and innovative approaches to personalized cancer therapy. The effort will include the creation of a consortium of five academic institutions—four in the United States and one abroad—to develop epigenetic therapies. Headquartered in Grand Rapids, MI, the Van Andel Institute is an independent research organization dedicated to discovery and science education.
According to GlobalData, a research and consulting firm in London, UK, the global market for biopsy devices will increase at a modest rate in the coming years, climbing from $506.4 million in 2013 to $602.4 million in 2019, a compound annual growth rate of 2.93%. However, the market for the biopsy devices will increase at much higher rates in countries with "emerging" economies, such as China, India, and Brazil. The firm suggested that greater scrutiny of routine screening for prostate and breast cancers is likely to lead to fewer biopsies, slowing sales.
A retrospective analysis of patients with oropharyngeal cancer who experienced disease recurrence after primary therapy found that those who were positive for human papillomavirus (HPV) had a higher overall survival rate than HPV-negative patients—54.6% vs. 27.6%, respectively—after two years. However, median time to progression was virtually the same for HPV-positive and HPV-negative patients—8.2 months vs. 7.3 months, respectively. The findings were presented at the 2014 Multidisciplinary Head and Neck Cancer Symposium in San Diego, CA, on February 20.
Basel, Switzerland–based Novartis announced that it will acquire the privately held biotechnology company CoStim Pharmaceuticals Inc., of Cambridge, MA, which focuses on immunotherapies for cancer, for an undisclosed sum. With the acquisition, Novartis adds late discovery stage immunotherapies aimed at several targets, including PD-1, to its pipeline.
Novartis also announced that its investigational oral compound LDE225 (sonidegib) met its primary endpoint—objective response rate within 6 months of treatment—in a phase II randomized, double-blind trial in patients with advanced basal cell carcinoma. Basal cell carcinoma accounts for more than 80% of non-melanoma skin cancers and can be life-threatening at advanced stages. The company said full study results will be presented at a future scientific meeting.
Astellas Pharma Inc. and Aveo Oncology terminated their agreement to develop Aveo's tivozanib for the treatment of breast and colon cancers. In December, the companies announced that the drug was not likely to meet its primary endpoint in a phase II colon cancer trial. Last month, they discontinued a phase II study in triple-negative breast cancer due to insufficient enrollment.
GlaxoSmithKline discontinued the manufacture and sale of Bexxar (tositumomab), a radio-immunotherapy with the radioactive isotope iodine-131 approved in 2003 for patients with CD20-positive relapsed or refractory non-Hodgkin lymphoma, due to relatively low profitability. Although studies have shown that the drug produced longer remissions than other therapies, it never gained widespread use, perhaps because the radioactive component made it more complicated to administer.
In a study published in Cancer Causes and Control, American Cancer Society researchers reported that there have been no significant increases in survival rates for Hispanics and non-Hispanic blacks with metastatic colon cancer, despite improvement in survival among whites, Asians, and people under age 65. The researchers analyzed data from nearly 50,000 patients from 1992 to 1997 and 2004 to 2009 across major ethnic groups and two broad age ranges. The findings, they say, underscore the need for concerted efforts to increase access to new treatments for minority groups and older patients, as well as a better understanding of the factors contributing to the disparities in survival.
Adolescent girls are less likely to engage in indoor tanning if they live in states with indoor-tanning laws, such as those that set age restrictions, according to a study published online by the American Journal of Public Health. The researchers say that these laws might be effective in reducing the health and economic burden of skin cancer.
U.S. Food and Drug Administration (FDA) Commissioner Margaret Hamburg, MD, traveled to India to meet with government officials and drug company executives about the quality of medical products they export to the United States, after some significant lapses were uncovered in recent years. "I am pleased that, as a global leader in the pharmaceutical and foods sectors, India will continue partnering with us to ensure that the companies exporting products to the U.S. are adhering to established quality standards," Hamburg wrote in a blog post. According to the FDA, India is the second largest provider of finished drug products to the United States.
The U.S. Food and Drug Administration (FDA) approved ibrutinib (Imbruvica; Pharmacyclics) for use in patients with chronic lymphocytic leukemia who have received at least one previous therapy. In November 2013, the drug was approved for the treatment of mantle cell lymphoma. The FDA had granted the drug an accelerated review for both indications.
Pharmaceutical giant Roche secured an injunction in an Indian court that prevents two companies from comparing their biosimilars to its breast cancer drug Herceptin (trastuzumab). The court ruled that Mylan and Biocon, which developed Hertraz and CANMab, respectively, cannot refer to Herceptin or its manufacturing process, safety, effectiveness, or sales when marketing their products. Mylan and Biocon received approval from Indian drug regulators last fall to market generic versions of Herceptin.
Salt Lake City, UT–based Myriad Genetics has reached a settlement in the patent-infringement lawsuit it brought against competitor Gene by Gene Ltd. in Houston, TX, in July 2013. Effective immediately, "Gene by Gene will cease selling and marketing diagnostic tests in North America that include analysis of the BRCA1 and/or BRCA2 genes as standalone tests or in conjunction with gene panels," Myriad announced. Gene by Gene can continue to offer tests that analyze the genes in other parts of the world.
Releasing its 2013 financial results, AstraZeneca announced that it will cut an additional 550 jobs as part of an ongoing restructuring effort, bringing the total number of job losses to 5,600. Competition from generic products has eaten into the company's profits, and the restructuring is expected to save the company about $1.1 billion a year by the end of 2016.
The FDA issued its first annual report to the U.S. Congress summarizing the major actions it took during 2013 to prevent or mitigate drug shortages in the country. The report notes that the agency worked with drug manufacturers to avoid 140 shortages between January 1 and September 30, but the report concludes that "drug shortages remain a significant public health issue in the United States and a top priority for the FDA."
In a study published in Cancer, researchers found that young women who were current or recent smokers and had been smoking a pack a day for at least 10 years had a 6% increased risk of estrogen receptor positive breast cancer, but that smoking was not related to a woman's risk of triple-negative breast cancer. Many recent studies have linked smoking to an increased risk of breast cancer, but few have examined the risks of developing different disease subtypes.
In nationwide poll from the University of Utah's Huntsman Cancer Institute in Salt Lake City, 34% of 1,202 respondents said they would not seek genetic testing to predict their chances of developing a hereditary cancer, even if the cost of testing wasn't an issue, due to concerns about employment and insurability. However, 35% of respondents would be "extremely or very likely to seek aggressive prophylactic or preventive treatment" if they had a family history of cancer and genetic testing indicated a predisposition to cancer. Poll results are available at The Annual Huntsman Cancer Institute Survey.
In a survey of 706 women ages 18 to 25 conducted by the University of North Carolina Lineberger Comprehensive Cancer Center in Chapel Hill, 45% had used a tanning bed during their lives and 30% had used one in the last year. Most of the tanning-bed users were aware of the health risks but did so anyway, citing improved appearance as the reason. The results were published in JAMA Dermatology.
Noting "an unprecedented opportunity to contribute to preventing millions of avoidable cancers and other conditions in men and women worldwide," the President's Cancer Panel issued a report calling for greater use of vaccines against human papillomavirus (HPV). The report states that only about one third of 13- to 17-year-old girls, and less than 7% of boys in the same age group, have received all three doses of the vaccine. Increasing vaccination rates to 80%, the report notes, would prevent 53,000 future cases of cervical cancer and thousands of other HPV-associated cancers.
Woonsocket, RI-based CVS Caremark said that it will discontinue sales of tobacco products at its more than 7,600 CVS/pharmacy stores across the country by October 1, making the company the first national pharmacy chain to take this step in support of the health of its customers. "Put simply, the sale of tobacco products is inconsistent with our purpose," said Larry Merlo, president and CEO of CVS Caremark. The move is expected to cost the company $2 billion in sales annually.
Japanese drug maker Eisai announced that its investigational drug lenvatinib produced highly statistically significant improvement in progression-free survival in patients with radioiodine-refractory, differentiated thyroid cancer compared with placebo in a phase III trial. Lenvatinib is an oral inhibitor of select receptor tyrosine kinases involved in angiogenesis and tumor proliferation, including VEGFR 1–3, FGFR 1–4, PDGFR-ß, KIT, and RET. Eisai plans to submit the drug for approval in the United States, Japan, and Europe.
Mylan Pharmaceuticals began marketing Hertraz (trastuzumab) in India. The drug is the world's first biosimilar to Herceptin (Roche/Genentech).
Due to insufficient enrollment, Aveo Oncology and Astellas Pharma have decided to discontinue a phase II trial of tivozanib in patients with locally recurrent or metastatic triple-negative breast cancer. The trial was evaluating the efficacy of tivozanib in combination with paclitaxel compared with a placebo plus paclitaxel. Last month, a phase II study of the drug in patients with colorectal cancer was stopped after an interim analysis concluded that the study was unlikely to meet its primary endpoint.
Thermo Fisher Scientific announced that it has completed the acquisition of Life Technologies for about $13.6 billion, plus the assumption of $1.5 billion in net debt. Last week, the U.S. Federal Trade Commission approved the deal upon the condition that Thermo Fisher sell certain assets related to siRNA reagents and cell cultures.
The International Cancer Genome Consortium announced that it is making data from more than 10,000 cancer genomes available to the scientific community through an Internet portal available at www.icgc.org. The data include the clinical characteristics of each tissue donor and functional information about the tumors, such as patterns in RNA expression, as well as the raw sequencing data. The information can be used by cancer researchers around the world to better understand the genomic basis of cancer, accelerate cancer research, and aid in the development of targeted treatments.
The American Association for Cancer Research (AACR) announced that it plans to open an office in China to better support and promote collaboration among researchers around the world. More than 30% of AACR's 34,000 members are located outside the United States, with 12% in Asia. AACR will elaborate on the plans at its 2014 annual meeting, which will be held in San Diego, CA, April 5–9.
Following in the steps of the NIH, as well as GlaxoSmithKline, Gilead Sciences, and other corporations, and bowing to pressure from activists, Merck is ending research on chimpanzees.
Pfizer announced that its experimental drug dacomitinib, an irreversible pan-HER kinase inhibitor, did not meet its goals in two separate phase III studies involving patients with non–small cell lung cancer (NSCLC). In one trial, patients previously treated with chemotherapy experienced no statistically significant improvement in progression-free survival compared with patients treated with erlotinib (Tarceva; Roche). Nor did the drug prolong overall survival compared with placebo in patients previously treated with chemotherapy and an EGFR tyrosine kinase inhibitor. A third phase III trial, comparing dacomitinib with gefitinib in treatment-naïve patients with EGFR-mutant advanced NSCLC, will continue.
According to an analysis of 7,776 studies registered in Clinicaltrials.gov, approximately 20% of the studies were not completed "for reasons that had nothing to do with the treatment's safety or effectiveness," said Matthew Galesky, MD, director of the Genitourinary Medical Oncology Program at the Tisch Cancer Institute in New York, NY. Lack of accrual was the most common cause of trials failing to be completed, and genitourinary cancer trials were no more likely to fail than trials in other cancer types. Galesky will present the findings on January 31 at the American Society of Clinical Oncology's Genitourinary Cancers Symposium.
Tightening domestic regulations and stronger measures to protect Indians who enroll in clinical trials seem to have become an impediment to research in the country. The Pharma Letter (London, UK) reported that in India in 2013, just 19 studies started, compared with 262 in 2012, a 93% drop.
Being frequently roused from sleep, as is the case in people with sleep apnea, seems to accelerate tumor growth, at least in mice. According to a study published in Cancer Research, mice that were awakened every 2 minutes over 7 days developed tumors that were twice as large as those in mice that slept regularly. The researchers noted that toll-like receptor 4 (TLR4), which helps control activation of the innate immune system, could be a potential drug target, because mice lacking TLR4 had tumor growth that was no greater than in mice with undisturbed sleep.
Overall career satisfaction among oncologists is high, especially for those in academic practice, according to a survey of 1,490 oncologists published in the Journal of Clinical Oncology. However, about 45% of respondents had at least one symptom of burnout, such as emotional exhaustion. Younger oncologists and those who spent the most hours seeing patients were more likely to experience symptoms of burnout.
"The checks and balances that once ensured scientific fidelity have been hobbled. This has compromised the ability of today's researchers to reproduce others' findings," wrote Francis Collins, MD, PhD, and Lawrence Tabak, DDS, PhD, director and principal deputy director of NIH, respectively, in Nature. The two then outlined steps that the agency plans to take, such as developing a training module for intramural postdoctoral fellows, to help improve reproducibility of research findings.
President Barack Obama signed a $1.1 trillion spending bill that will fund the federal government through fiscal year 2014, which ends on September 30. The bill includes $29.9 billion for the NIH, about $1 billion more than in 2013 after sequestration. However, that sum is still more than $710 million short of its original allotment for 2013.
About 5.6 million children alive today—or one out of every 13 children under age 18—will die prematurely from smoking-related disease unless current smoking rates drop, according to a report from Acting Surgeon General Boris Lushniak, MD, MPH. It also concludes that cigarette smoking kills nearly 500,000 Americans a year, with 16 million more suffering from smoking-related conditions, costing the country $289 billion annually. The report comes 50 years after the historic 1964 Surgeon General's report concluded that cigarette smoking causes lung cancer.
Biocon will begin selling a cheaper version of Roche's Herceptin (trastuzumab), a treatment for breast cancer, in India next month. The Bangalore-based company will sell its drug, called CANMab, for about 25% less than Herceptin, but the retail price will still be out of reach for most women. Last year, Roche stopped defending its patent on Herceptin in India after the government there said it might issue a compulsory license to let others develop the drug in an effort to bring down prices.
Pharmaceutical giant Novartis announced that it will cut 500 jobs as part of its ongoing reorganization. The cuts will affect support positions at the company's headquarters in Basel, Switzerland, as well as operational roles in pharmaceutical development.
The $1,000 human genome sequence is now a reality, according to San Diego, CA–based Illumina, Inc., making mass sequencing possible. The company's CEO, Jay Flatley, introduced its HiSeq X sequencer, designed to process 20,000 genomes a year at a cost of $1,000 each, at the JP Morgan Chase & Co. health care conference in San Francisco, CA.
In a study published in the Annals of Oncology, researchers conclude that Europe may not have enough medical oncologists to keep pace with the rising toll of cancer. The report shows that 12 countries, mostly in Western Europe, will probably have enough medical oncologists over the next 8 years, but researchers were not able to gather sufficient information from 15 other European Union members to assess their needs. The European Society for Medical Oncology warned that the lack of information "must be urgently addressed."
Adding cetuximab (Erbitux; Bristol-Myers Squibb and Lilly) to standard chemoradiation with cisplatin and paclitaxel yielded no survival benefit for patients with esophageal cancer, researchers announced at the American Society of Clinical Oncology's Gastrointestinal Cancer Symposium. Patients in the cetuximab arm had a 2-year survival rate of 44%, versus 41.7% for patients in the control arm.
Also at the Gastrointestinal Cancer Symposium, Carmen Allegra, MD, of the University of Florida in Gainesville, reported that neoadjuvant chemoradiation with oral capecitabine (Xeloda; Roche) worked just as well as 5-fluorouracil infusion therapy for 1,600 patients with stage II and III rectal cancer in a clinical trial. "This study establishes capecitabine as the standard of care in the preoperative rectal setting," he said.
GlaxoSmithKline said the U.S. Food and Drug Administration (FDA) has given its drug Tafinlar (dabrafenib) a Breakthrough Therapy designation for lung cancer based on interim results from an ongoing phase II study. The designation means that the FDA will expedite its review of the drug.
Peregrine Pharmaceuticals said that it has received Fast Track designation from the FDA for its immunotherapy bavituximab, which is under investigation as a second-line treatment for non–small cell lung cancer. A phase III, randomized, double-blind trial comparing bavituximab plus docetaxel with a placebo plus docetaxel in patients with the disease is currently under way.
Cambridge, MA-based Cerulean Pharm announced that its CRLX101 reached its primary efficacy endpoint in an ongoing single-arm, phase II clinical trial in relapsed ovarian cancer, with at least four of 29 patients achieving 6 months of progression-free survival. CRLX101 inhibits topoisomerase 1 and hypoxia-inducible factor-1a. The company expects to present final data from the trial at the 2014 annual meeting of the American Society of Clinical Oncology.
The FDA approved Tafinlar for use in combination with Mekinist (trametinib) to treat patients with inoperable or metastatic melanoma with BRAF V600E or V600K mutations detected by an FDA-approved test. Both drugs were approved in 2013 for use as single agents in these patients.
APG101 significantly improved overall survival in patients with recurrent glioblastoma who have an epigenetic biomarker associated with the CD95 ligand, the target of APG101, according to German drugmaker Apogenix. Median overall survival of patients in the phase II trial who received APG101 plus radiotherapy was 16.1 months compared with 6.5 months in patients treated with radiotherapy alone. Thomas Hoeger, CEO of Heidelberg-based Apogenix, presented the data at the Biotech Showcase in San Francisco, CA.
Between 2005 and 2009, lung cancer incidence rates have dropped by 2.6% per year among men and 1.1% per year among women, according to a report from the Centers for Disease Control and Prevention. Among adults ages 35 to 44, the drop in incidence rates was even greater—6.5% per year among men and 5.8% per year among women. In all age groups, lung cancer incidence rates dropped more quickly among men than among women.
Ludwig Cancer Research of New York, NY, gave $540 million to fund cancer research at the six U.S. Ludwig Centers, which are located at Harvard University, Johns Hopkins University, the Massachusetts Institute of Technology, Memorial Sloan-Kettering Cancer Institute, Stanford University, and the University of Chicago. Ludwig has now given a total of $900 million to these institutions—and $2.5 billion globally—to advance cancer research.
Pfizer and The University of Texas MD Anderson Cancer Center in Houston announced that they will collaborate to develop immune-based approaches to cancer treatment. The 3-year agreement is designed to help speed progress on immune-based treatments and to more efficiently identify and exploit new combination therapies, as well as biomarkers to guide and monitor treatment.
Researchers at the Cleveland Clinic in Ohio have developed an online tool to estimate a patient's risk of colorectal cancer. Dubbed CRC-PRO and available at rcalc.ccf.org, the calculator can help physicians determine when screening is needed and help reduce health care costs by cutting down on unnecessary testing, the researchers say.
In a review published in the New England Journal of Medicine, researchers wrote that tripling taxes on cigarettes worldwide would reduce the number smokers by one third and prevent 200 million premature deaths from lung cancer and other diseases in this century.
The U.S. Food and Drug Administration granted Fast Track designation to Peregrine Pharmaceuticals' investigational immunotherapy bavituximab for second-line treatment of non–small cell lung cancer (NSCLC). The company is currently conducting a 600-patient, phase III study comparing bavituximab plus docetaxel with a placebo plus docetaxel in NSCLC.
Expressing support for data-sharing efforts, Sanofi announced that its companies will provide access to clinical trial data and related documents for studies it conducts in people, although with some caveats. The studies must have been submitted to regulatory authorities in the United States and the European Union, and the products studied must have been approved by both agencies on or after January 1, 2014. Requests for access to clinical trial data can be made through clinicalstudydatarequest.com.
Researchers estimate that tobacco control efforts in the United States have prevented about 8 million premature deaths, with those people each gaining about 20 years of life on average. According to a second study, although the percentage of the population that smokes daily has decreased, the number of cigarette smokers worldwide has increased due to population growth. The studies were published in a special issue of the Journal of the American Medical Association to mark the 50th anniversary of the surgeon general's first report on cancer and smoking.
The U.S. Preventive Services Task Force (USPSTF) published its guidelines on screening for lung cancer, recommending annual low-dose computed tomography for people from 55 to 80 years old who are at high risk of the disease—those who have smoked a pack of cigarettes a day for 30 years and currently smoke or have quit within the past 15 years. Screening should be discontinued once a person has not smoked for 15 years or develops a health problem that substantially limits life, the guidelines say.
The USPSTF also released its recommendations for genetic testing for BRCA1 and BRCA2 mutations, saying that the vast majority of women do not need to be tested. The panel advised women with a strong family history of breast, ovarian, tubal, or peritoneal cancer to talk to their doctors about their risk and the possibility of genetic testing.
The city council in New York City, NY, voted to expand its ban on indoor smoking to include e-cigarettes, battery-operated devices that deliver nicotine vapor to users. Several states, including Arkansas, New Jersey, North Dakota, and Utah, have already added e-cigarettes to their indoor smoking bans.
The U.S. Food and Drug Administration (FDA) granted priority review to the supplemental biologics license application from GlaxoSmithKline and Genmab for the use of Arzerra (ofatumumab) in combination with an alkylator-based therapy to treat patients with chronic lymphocytic leukemia (CLL) who have not received prior therapy and are not candidates for fludarabine-based therapy. A human monoclonal antibody, Arzerra is approved for the treatment of patients with CLL refractory to fludarabine and alemtuzumab.
Atossa Genetics of Seattle, WA, submitted a premarket 510(k) notification with the FDA seeking approval for its ForeCYTE breast cancer test device, which includes a breast pump and kit to collect nipple aspirate fluid for laboratory cytology testing.
Ariad Pharmaceuticals of Cambridge, MA, received FDA approval to begin marketing Iclusig (ponatinib) again, although to a smaller population of leukemia patients. Sales of the drug, originally cleared in 2012, were halted in October when regulators noticed an increased incidence of blood clots.
Pharmaceutical giant Bayer agreed to buy Algeta, its partner on the prostate-cancer medicine Xofigo (radium-223 dichloride), for about $2.9 billion to gain control of the drug and experimental radiation therapies. Xofigo, which binds to minerals in bone to deliver radiation to bone tumors, is approved for use in patients with metastatic, castration-resistant prostate cancer.
In a Christmas Eve opinion piece in The Washington Post about funding biomedical research, NIH Director Francis Collins, MD, PhD, wrote, "The economic benefits of NIH funding include a return-on-investment of research grants to local economies and cost savings from decreased disease burdens. But beyond that, for the millions of sick people awaiting treatments and cure, the investment in NIH's mission is priceless. If an investment in hope is not worth supporting, I don't know what is."