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Rescuing and 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 13 years. It is crucial to advance strategies to reduce this time frame, decrease costs and improve success rates.

Drug rescue and repurposing are two such strategies. Drug “rescue” refers to research using small molecules and biologics that previously were used in studies but not further developed and submitted for Food and Drug Administration (FDA) approval. “Repurposing” generally refers to studying a small molecule or a biologic approved by the FDA to treat one disease or condition to see if it is safe and effective for treating other diseases. (More about the need for drug rescue and repurposing)

woman researcher looking in microscope in lab

Many rescued compounds and approved drugs for other uses already have been tested in humans, so detailed information is available on their pharmacology, formulation and potential toxicity. Because rescue and repurposing build upon previous research and development efforts, new candidate therapies could be ready for clinical trials quickly, hastening their review by the FDA and, if approved, integrated into health care.

Bringing Together the Key Players

Exploring New Uses for Abandoned and Approved Therapeutics

In April 2011, NIH convened an NIH-Industry Roundtable that brought together senior leaders and experts from the pharmaceutical industry, government, academia and the nonprofit sector to explore opportunities to foster new partnerships that could facilitate drug rescue and repurposing. Participants identified several challenges and opportunities in exploring new uses for these compounds.

Pilot Program Tests New Therapeutic Uses

In May 2012, NCATS launched a collaborative program that matches NIH-funded researchers with a selection of pharmaceutical molecular compounds 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 compounds that already have cleared several key steps along the way.

Through this innovative collaboration, several pharmaceutical companies have made some of their high-quality compounds 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. With funding support from the NIH Common Fund, NCATS announced the pilot program awards in June 2013.

NCATS Pharmaceutical Collection

NPC logoAlready, NCATS 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 NCATS’ Pharmaceutical 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.

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.