New $20 Million NIH Program Aims to Uncover New Uses for Existing Drugs

NIH's Translational Science Institute Creates New Program To "Teach Old Drugs New Tricks"

-- The initiative, Discovering New Therapeutic Uses for Existing Molecules, matches researchers with a selection of pharmaceutical industry compounds to help scientists explore new treatments for patients --

U.S. Secretary of Health and Human Services Secretary Kathleen Sebelius announced today a new $20 million grant program at the National Institutes of Health (NIH) to investigate whether existing drug compounds can be used to treat diseases and illnesses other than those they were originally developed to treat.  At today's press conference, Sebelius said the program is designed to "see if we can teach old drugs new tricks."

The pilot phase of this new public-private partnership is a collaboration between NIH's newest institute – the National Center for Advancing Translational Sciences (NCATS) – and pharmaceutical companies AstraZeneca, Eli Lilly, and Pfizer.  These three companies are offering access to dozens of drug compounds (and related data) that have cleared regulatory and toxicity hurdles but may not have proven effective for the specific use for which they were developed.

The program is intended to provide $20 million in grants in Fiscal Year (FY) 2013, with the hopes of more than $20 million in both FY 2014 and FY 2015.  NIH expects to award its first round of grants for pre-clinical and clinical feasibility studies in the spring of 2013. 

National Institutes of Health (NIH) Director Francis Collins expressed excitement about NIH's role as matchmaker between investigators and the private sector.  He explained that the $20 million in FY 2013 funding is intended to be catalytic – meaning if and when an investigator is able to validate his or her work and reach clearly defined milestones, other NIH institutes will have the opportunity to kick in funding to further the work.

As an example of what this program is intended to accomplish, patient advocate Michael Manganiello, diagnosed in 1987 with HIV, spoke about azidothymidine (AZT), which was shown to be an ineffective cancer treatment but later proved to be the first medicine effective against HIV.  Another example given by NIH leadership was thalidomide, developed to treat morning sickness, but now being used to treat multiple myeloma.

"The idea here is, let's not depend on serendipity [in finding new uses for existing compounds]; let's do this in a more systematic, comprehensive way by going from individual ideas to the crowd-source model," Collins said.

PAN's Government Relations Director, Becca O'Connor, thinks this is exciting news for the Parkinson's community.

“Parkinson’s is still generally being treated with the same symptomatic drug treatment that was discovered 40 years ago,” said O'Connor.  "Creating public-private partnerships like the one announced today shows great initiative and innovation on the part of NIH, and we are excited for the promise these potential new discoveries might yield.  We're also encouraged to hear that NIH is open to other pharmaceutical companies joining the program."

O'Connor added, "Now more than ever it is important to fight for robust FY 2013 funding for NIH to ensure innovative programs like the one announced today get the dollars they need.  For years, PAN has been at the forefront of championing the importance of translational research, and the fact that NCATS can announce something like this only four months into the Institute's existence is remarkable and, hopefully, a sign of more great things to come."

How will potential new findings be managed?  According to NIH, "The pilot program incorporates innovative template agreements designed to streamline the legal and administrative process for participation by multiple organizations.  These template agreements reduce time, cost, and effort, as well as allow greater participation than traditional partnerships.  The templates also provide a roadmap for handling intellectual property used in or developed through the program.  Participating industry partners will retain the ownership of their compounds, while academic research partners will own any intellectual property they discover through the research project with the right to publish the results of their work."

Media coverage, thus far, of this announcement includes:
Bloomberg:  Pfizer, Lilly Help U.S. Test Failed Drugs for New Illness
Washington Post:  Collaboration seeks to find new uses for failed drugs
Reuters:  U.S. to partner with Big Pharma for drug discovery

NIH Press Release:
NIH launches collaborative program with industry and researchers to spur therapeutic development

NIH Radio Program about the announcement:  Listen here

Learn more about PAN's role in the creation of NCATS:  Click here