Teaching Old Drugs New Tricks

Under a pilot program announced by NIH last week, dozens of drug compounds that have advanced to clinical studies but are no longer being pursued for their original indication will be available to NIH-funded researchers. The program provides research grant support and template agreements with the pharmaceutical firms that own the compounds.

The pilot program—Discovering New Therapeutic Uses for Existing Molecules—is the first major endeavor of the National Center for Advancing Translational Sciences (NCATS), which was established this year with a budget of $575 million. The collaborative program initially involves three pharmaceutical partners—Pfizer, AstraZeneca, and Eli Lilly & Co.—which have agreed to make 24 compounds available.

“To accelerate our nation’s therapeutic development process, it is essential that we forge strong, innovative, and strategic partnerships across government, academia, and industry,” Department of Health & Human Services Secretary Kathleen Sebelius said at the May 3 rollout of the program. She added that this pilot is effectively the “crowdsourcing” of pharma compounds to the brightest drug development minds in the country.

The compounds from the pharmaceutical partners are about halfway through the development pipeline, having been studied for five to seven years with tens of millions of dollars invested in them. They have cleared the safe-for-human-use step.

Interested researchers can begin applying for NCATS grants beginning next month. The applications will be peer reviewed. NCATS will dedicate $20 million to the program in fiscal 2013 to fund awards.

NIH and NCATS are playing a matchmaking role in this process, explained NIH Director Francis S. Collins at the rollout. The goal is to create new opportunities for drugs that failed their intended use, a redirection that’s happened serendipitously. “We must generate more success stories in a systematic way,” he said.

For their part, the drug firms have committed to supply researchers with the compounds and related data.

“Pfizer has a rich history of partnering with the academic and public sectors to discover and develop innovative medicines,” said Rod MacKenzie, head of Pfizer PharmaTherapeutics R&D. “We believe that participating in this unique program, and making well-researched molecules available to NIH’s broad network of researchers, is another important step in advancing a vibrant biomedical ecosystem.”