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Repurposing Drugs

Discoveries about the molecular basis of disease provide unprecedented opportunities to translate research findings into new medicines. However, developing a brand-new drug takes an enormous amount of time, money and effort, mainly because of bottlenecks in the therapeutic development process. Delays and barriers can mean that translation of a promising molecule into an approved drug often takes more than 14 years. It is crucial to advance strategies to reduce this time frame, decrease costs and improve success rates.

Drug repurposing is one such strategy. “Repurposing” generally refers to studying a compound or biologic (referred to as agents) to treat one disease or condition to see if it is safe and effective for treating other diseases. 

woman researcher looking in microscope in lab

Many agents approved for other uses already have been tested in humans, so detailed information is available on their pharmacology, formulation and potential toxicity. Because repurposing builds upon previous research and development efforts, new candidate therapies could be ready for clinical trials quickly, speeding their review by the Food and Drug Administration and, if approved, integration into health care.

Testing New Therapeutic Uses

In May 2012, NCATS launched a collaborative program that matches NIH-funded researchers with a selection of pharmaceutical agents to help scientists explore new treatments for patients. The Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program aims to improve the complex and time-consuming process of developing new treatments and cures for disease by finding new uses for agents that already have cleared several key steps along the development path.

Through this innovative collaboration, several pharmaceutical companies have made some of their high-quality agents available for research. By making these resources broadly available, NIH and its partners aim to stimulate the development of therapies for diseases that still lack effective treatments. New funding opportunities now are open.

NCATS Pharmaceutical Collection

NPC logoNCATS is enhancing repurposing activities to organize available data on drugs and investigational compounds through the NCATS Pharmaceutical Collection. This resource, published in the April 27, 2011, issue of Science Translational Medicine, is a publicly accessible database of small molecule compounds that have been approved by regulatory agencies from the United States, Canada, Europe and Japan as well as all compounds that have been registered for human clinical trials.

In March 2012, NCATS and Eli Lilly and Company agreed that the collection of 3,800 approved and investigational medicines would be screened using Lilly’s state-of-the-art Phenotypic Drug Discovery (PD2) panel. Comprehensive knowledge of the biological profiles of these medicines and molecules may enable biomedical researchers to better predict treatment outcomes, improve drug development, and lead to more specific and effective approaches.

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New Therapeutic Uses

Freda Lewis-Hall, M.D.

In this video, Freda Lewis-Hall, M.D., chief medical officer at Pfizer, discusses the Discovering New Therapeutic Uses for Existing Molecules program.