Dr. Brian Dayton, September 12, 2013

Kudos to the National Institutes of Health for putting some monetary teeth behind its commitment to drug repurposing.  In June of this year, NIH awarded $12.7 million to match nine academic research groups with a selection of existing compounds from pharmaceutical industry partners to explore new treatments for patients in eight disease areas, including Alzheimer's, Duchenne muscular dystrophy and schizophrenia.  Led by the National Center for Advancing Translational Sciences (NCATS), this pilot program, called Discovering New Therapeutic Uses for Existing Molecules, provides academic research groups with milestone-driven, cooperative agreement awards valid for two or three years.

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According to NCATS' New Therapeutic Uses program, they hope to shorten “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.”  This program continues to demonstrate the commitment NIH has to repurposing as first laid out in Director Collins’ 2011 article in Nature Review Drug Discovery, and is a direct outcome of a 2011 NIH-Industry Roundtable workshop aimed at fostering new collaborations similar to those represented by this initiative.

We applaud the NIH on its efforts to provide a more efficient route to new treatment options for patients.  This path can be shortened even more efficiently by repurposing those drugs which are already approved for use by the FDA.  By expanding the armamentarium of validated off-label uses of approved drugs, it is our desire to bring new hope to those suffering even more quickly.

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