National Institutes of Health Funds Projects to Identify New Uses for Existing Compounds

The National Institutes of Health (NIH) has awarded $12.7 million to match nine academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, the NIH announced in a press release. The collaborative pilot initiative, Discovering New Therapeutic Uses for Existing Molecules, is led by the National Center for Advancing Translational Sciences (NCATS) and is funded by the NIH Common Fund.

The initiative aims to use existing, partially developed compounds that could be advanced to clinical trials more quickly than starting from scratch. “With thousands of diseases remaining untreatable, there is a sense of urgency to accelerate the pace at which discoveries are transformed into therapies for patients,” said Health and Human Services secretary Kathleen Sebelius in the press release. “This program helps forge partnerships between the pharmaceutical industry and the biomedical research community to work together to tackle problems that are beyond the scope of any one organization or sector.”

AbbVie (formerly Abbott), AstraZeneca, Bristol-Myers Squibb, Eli Lilly and Company, GlaxoSmithKline, Janssen Research & Development, Pfizer, and Sanofi are participating in the pilot phase of the program.

NCATS launched this initiative in 2012 to help re-engineer the research pipeline using an innovative strategy to identify new uses for compounds that have undergone significant research and development by industry, including safety testing in humans. The center crowdsourced the industry compounds to academic researchers nationwide to gain ideas for new therapeutic uses with the ultimate goal of developing new treatments for patients. The program also tested newly created template agreements, which enabled negotiations to be completed in fewer than 11 weeks, versus a typical timeline of a year or more.

“Public-private collaborations are crucial for successful translation; no one organization can succeed alone,” said NCATS director Christopher P. Austin, MD. “This initiative has created a marketplace to connect academic researchers with potential new drugs, as well as template agreements that streamline the process by limiting the amount of negotiation required before a project can begin.”

Each award recipient will test a selected compound for its effectiveness against a previously unexplored disease or condition. The eight disease areas represented are alcohol dependence, Alzheimer’s disease, calcific aortic valve stenosis (a condition in which the heart valve hardens and makes it difficult to pump blood out of the heart), nicotine dependence, peripheral artery disease, schizophrenia, and two rare diseases: Duchenne muscular dystrophy and the progressive lung disease lymphangioleiomyomatosis.

These cooperative agreements will fund projects for researchers to conduct pre-clinical validation and additional safety studies as needed. If specific milestones are met, clinical feasibility studies or proof-of-concept clinical trials will be initiated to test whether the selected compounds may be effective as treatments for other diseases. The projects will be supported for up to three years.