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Crowdsourcing initiative will enable exploration of potential treatments in eight disease areas

The National Institutes of Health 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 Alzheimers disease, Duchenne muscular dystrophy and schizophrenia. The collaborative pilot initiative, called Discovering New Therapeutic Uses for Existing Molecules, is led by the National Center 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 percent. This failure rate means, however, that many existing partially developed compounds 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. 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.

Innovative, collaborative approaches that improve the therapeutic pipeline are crucial for success, said NIH Director Francis S. Collins, M.D., 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 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, M.D. 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, M.D., Ph.D., Kennedy Krieger Institute, Baltimore, talks about Duchenne muscular dystrophy and how the New Therapeutic Uses program can make a difference in potentially accelerating a treatment for this devastating disorder.

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