A revolutionary new cancer treatment has been approved by the Food and Drug Administration (FDA).
Kymriah is the first gene therapy for leukemia available in the United States.
It is a CAR-T (chimeric antigen receptor T cell) immunotherapy, designed to treat acute lymphoblastic leukemia (ALL), a serious form of cancer in children and young adults.
ALL is a potentially fatal cancer of the blood and bone marrow.
The National Cancer Institute estimates there are 3,100 people under the age of 20 diagnosed each year.
CAR-T therapy was pioneered by Dr. Carl June at the University of Pennsylvania, who has been working on the treatment for almost three decades.
How Kymriah works
Unlike other current cancer treatments such as surgery, chemotherapy, and radiation, Kymriah uses the body’s own immune system to fight against the cancer.
Kymriah works through a process of collecting a patient’s cells — specifically T cells that work as part of the immune system — and genetically modifying them to recognize and attack cancer cells.
The modified T cells are engineered to include chimeric antigen receptors, which target leukemia cells.
Once this process has been completed, the modified cells are infused back into the patient’s body.
“[The] approval is transformational for pediatric cancer patients and cancer research overall. Using gene therapy to reprogram and educate a patient’s own immune cells to target, attack, and eliminate cancer is a brilliant approach, and we are very encouraged that it has been approved by the FDA for children with acute lymphoblastic leukemia (ALL),” Ramy Ibrahim, vice president of clinical development at the Parker Institute for Cancer Immunotherapy, told Healthline.
A costly treatment
The procedure is personalized for the individual.
As such, it has limitations on the price and scope of its manufacturing.
The therapy is being licensed and brought to market by the pharmaceutical company Novartis.
The company plans to make Kymriah available to about 30 hospitals by the end of the year.
The price of the therapy is tagged at around $500,000.
It also comes with some serious potential risks.
CAR-T can lead to a life-threatening condition known as cytokine release syndrome, as well as severe neurological events.
So, hospitals and clinics that offer Kymriah will have to have special certifications.
For now, it is only approved for use on ALL. It can’t be used to treat any of the myriad other deadly forms of cancer... yet.
However, the approval of Kymriah bodes well for the future of gene therapy to treat other forms of cancer.
‘Beginning of the journey’
Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society, is excited by the drug’s approval, but he has some reservations.
“This is not the final answer to cancer treatment. This is elegant science. The researchers have done outstanding work. There is still much more work to do,” Lichtenfeld told Healthline.
For now, Kymriah is only available to a small subsection of cancer patients. Even then, it is prescribed only as a last resort when other forms of treatment have failed.
“We’re at the beginning of the journey,” said Lichtenfeld. “We’re far from the end.”