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Discovering New Therapeutic Uses for Existing Molecules

Researcher using a pipette and a microscope

Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) is a collaborative pilot program designed to develop partnerships between pharmaceutical companies and the biomedical research community to advance therapeutics development. This innovative program matches researchers with a selection of molecular compounds from industry to test ideas for new therapeutic uses, with the ultimate goal of identifying promising new treatments for patients.

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.

Launched in May 2012, NCATS' New Therapeutic Uses program helps 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. By using compounds that already have cleared several key steps in the development process, scientists nationwide have a strong starting point to contribute their unique expertise and accelerate the pace of therapeutic development.

AbbVie (formerly Abbott), AstraZeneca; Bristol-Myers Squibb Company; Eli Lilly and Company; GlaxoSmithKline; Janssen Research & Development, LLC; Pfizer; and Sanofi provided 58 compounds for the pilot program. NIH then turned to academic researchers nationwide, crowdsourcing for ideas to find new uses for these partially developed compounds.

Funding Information

In June 2013, NIH awarded $12.7 million to fund nine projects through this NCATS-led pilot program. These milestone-driven, cooperative agreement awards will be valid for two or three years and pair academic research groups with a selection of pharmaceutical industry compounds to explore new treatments for patients in eight disease areas, including Alzheimer's, Duchenne muscular dystrophy and schizophrenia. The NIH Common Fund provides funding for the pilot phase of these awards.

Scientific expertise will be provided by seven other NIH Institutes and Centers, including the National Cancer Institute; National Heart, Lung, and Blood Institute; National Institute on Aging; National Institute of Alcohol Abuse and Alcoholism; National Institute on Drug Abuse; National Institute of Mental Health; and National Institute of Neurological Disorders and Stroke.

The awards were made in response to RFA-TR-004 and to RFA-TR-12-005. Learn more on the Funding Information page.

Library of Agents

The New Therapeutic Uses library of industry-provided agents features compounds and biologics that have undergone significant pre-clinical and Phase I testing and are ready for additional testing in humans.

Template Agreements

triangle logoTo facilitate and speed collaboration, NIH developed template agreements designed to streamline the legal and administrative process for participation by multiple organizations. These agreements help facilitate complex negotiations among all parties involved in the program, enabling the research to begin faster.

Related Links

Contact

Christine Colvis, 301-451-3903


For More Information

News Release

Fact Sheet

2013 Project Awards

Program Videos

Program Audio Clips

FAQ About New Therapeutic Uses

Funding Information

Library of Agents


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