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, including Alzheimer’s disease, Duchenne muscular dystrophy and schizophrenia. The collaborative pilot initiative, called Discovering New Therapeutic Uses for Existing Molecules, is led by the National Centre for Advancing Translational Sciences (NCATS) and funded by the NIH Common Fund.

The process of developing a new therapeutic is long and difficult. The average length of time from target discovery to approval of a new drug is more than 13 years, and the failure rate exceeds 95 per cent. This failure rate means, however, that many existing partially developed compounds could be advanced to clinical trials more quickly than starting from scratch.

Dr Jordan Miller, Ph.D., Mayo Clinic, Rochester, Minnesota, discusses his team’s research effort to test the safety of an investigational drug in heart valve disease patients in slowing progression of stenosis, an abnormal narrowing of vessels or other structures.

“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. “This programme 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.”

“Innovative, collaborative approaches that improve the therapeutic pipeline are crucial for success,” said NIH director Francis S Collins, MD, Ph.D. “This unique collaboration between academia and industry holds the promise of trimming years from the long and expensive process of drug development.”

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

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 programme 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.”

Dr Kathryn Wagner, MD, Ph.D., Kennedy Krieger Institute, Baltimore, talks about Duchenne muscular dystrophy and how the New Therapeutic Uses programme can make a difference in potentially accelerating a treatment for this devastating disorder.

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.

The NCATS is a distinctly different entity in the research ecosystem. Rather than targeting a particular disease and fundamental science, NCATS focuses on what is common across diseases and the translational process. The Centre emphasizes innovation and deliverables, relying on the power of data and new technologies to develop, demonstrate and disseminate improvements in translational science.

The NIH Common Fund encourages collaboration and supports a series of exceptionally high-impact, trans-NIH programmes. Common Fund programmes are designed to pursue major opportunities and gaps in biomedical research that no single NIH Institute could tackle alone, but that the agency as a whole can address to make the biggest impact possible on the progress of medical research.

NIH, the nation's medical research agency, and is a component of the US Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.