WASHINGTON -- The National Institutes of Health (NIH) announced Thursday that it will partner with three drug companies -- Pfizer, AstraZeneca, and Eli Lilly -- to make data on two dozen already researched, but mostly discarded, molecular compounds available to academic researchers in hope of developing new disease treatments.
The scale of the partnership is unprecedented, according to Kathy Hudson, PhD, acting deputy director of the NIH's newly created National Center for Advancing Translational Science (NCATS), which is in charge of the collaboration.
Under the pilot collaboration, the drug companies will make available all the research data on compounds that stemmed from years of research and millions of dollars. The 24 compounds have already been tested in humans, meaning there is detailed information available for dosing, pharmacology, and toxicity.
The compounds at one time held promise as disease treatments, but research on them has since sputtered and in some cases stalled.
Yet those compounds could prove to be treatments for other diseases.
"Many of the compounds may have other potential uses; we just need to do the research to identify them," Health and Human Services Secretary Kathleen Sebelius told reporters.
The compounds would likely be used for research on any of 4,500 diseases for which the genetic causes are now known, thanks to the mapping of the human genome.
Many of those diseases are rare diseases, said NIH director Francis Collins, MD. Some compounds could provide clues to treatments for neurological diseases as well, he said.
Rod MacKenzie, PhD, head of pharmatherapeutics research and development at Pfizer, agreed about that the compounds may hold answers for neurological diseases. "If I had a wish, it would be we'd find something that would lead to a breakthrough in Alzheimer's disease," MacKenzie said during the press conference.
Collins pointed to a few compounds that started off as potential treatments for one disease, didn't work, and then were found to be effective for a different disease.
One prime example: azidothymidine, or AZT, which was originally developed as a cancer treatment. It wasn't effective in treating cancer, but it was later found to be the first effective medicine for HIV.
Or the drug thalidomide, which was developed to treat morning sickness, but was pulled from the market in the early 1960s after it was found to cause birth defects. However, it was later found to be an effective treatment for multiple myeloma.
Under the collaboration, researchers -- either in the private industry, in academia, or with the government -- could apply for an NIH grant to investigate a given compound provided by any one of the drug companies.
If a researcher discovered an effective treatment for a disease or condition using a compound developed by a drug company, the researcher would have intellectual property rights, and the company would still own the rights to the drug. (The government would not get royalties from the findings, except if the investigator worked for NIH.)
It take an average of 14 years and $2 billion to move a drug from lab to clinic, Collins said.
The compounds being included in the pilot collaboration are about halfway through a traditional development program, so the drug companies have invested between 5 and 7 years and tens of millions of dollars in their development, MacKenzie said.
Hudson said the government is not releasing the list of specific compounds that will be available for another month, when it will make an announcement about how researchers can apply for the grants.
President Obama's proposed fiscal 2013 budget calls for $575 million for NCATS, which will house the collaboration. Of that proposed budget, $20 million in 2013 would go toward grants for academic scientists to perform research on the compounds provided by drug companies, Hudson said. The program would be funded for at least $20 million annually in subsequent years.
NIH said it hopes to get other drug companies to join the collaboration. The center also plans to run projects that flow in the other direction, in which NIH would perform preclinical groundwork on promising compounds and then hand off the information to pharmaceutical companies that would refine the compounds into new drugs.