NIH Announces Nine Projects to Repurpose Old Drugs

U.S. National Institutes of Health

The National Institutes of Health (NIH) today unveiled the winners of an unusual competition in which academic researchers teamed up with pharmaceutical companies to propose new uses for abandoned drugs. The nine projects, funded at a total of $12.7 million a year, show that NIH's 19-month-old National Center for Advancing Translational Sciences (NCATS) is achieving its goal of finding new ways to speed drug development, NCATS officials said.

The program, called Discovering New Therapeutic Uses for Existing Molecules, aims to help companies work with academic researchers to repurpose drugs that passed safety testing but didn't help patients with the intended disease or were dropped for business reasons. A year ago, eight companies agreed to contribute 58 compounds and share data about them. NCATS invited researchers to submit ideas for developing the drugs, and the agency received about 160 preapplications. NCATS then linked up the investigators and companies to hone the strongest proposals and flesh out template legal agreements. The NIH then reviewed full applications, nine of which made the final cut.

The projects will study seven of the 58 compounds. The research will include animal studies and some early clinical trials for eight diseases, including Duchenne muscular dystrophy, alcohol and nicotine addiction, and common disorders like Alzheimer's. "We can say with great confidence that the crowdsourcing of potential diseases these molecules might treat … did exactly what we hoped," said NCATS Director Christopher Austin during a teleconference today. NCATS has identified only some of the compounds in its announcement in order to protect the companies' intellectual property.

Don Frail, a vice president at AstraZeneca, which will contribute two compounds for three of the projects, says that the effort has already disproved skeptics who doubted that academic researchers could generate ideas that companies hadn't thought of already. "To me this creates unmatched partnerships," Frail says. "I think all of those who have participated have been quite pleased."

Austin says that NCATS now hopes to expand the pilot program, but that will depend on NIH funding—the agency's budget was cut 5% in 2013 due to sequestration and could face more trims next year.