Drug repurposing NIH // Drug Repurposing

Drug repurposing NIH

woman researcher looking in microscope in labDiscoveries 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.

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.

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Nature editorial: Repurposing drugs

by Drosophila3


Nature 465, 267–268 (20 May 2010) doi:10.1038/465267b
Published online 19 May 2010
The United States should protect investments used to find new uses for old drugs.
In 2007, a paper in the journal Cancer Cell announced that the compound dichloroacetate (DCA) had been found to shrink tumours in rats (S. Bonnet et al. Cancer Cell 11, 37–51; 2007). That news by itself would not have created much of a stir: many compounds tested in rodents raise hopes of their becoming potential cures, and almost as many go on to fail in human clinical trials

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