, May 2, 2011  —  In an effort to find new therapies for 6,000 rare diseases, National Institutes of Health researchers are screening drugs approved for other uses. They’re hoping to find off-label uses for the diseases afflicting some 25 million Americans.

“This is a critical step to explore the full potential of these drugs for new applications,” said Dr. Francis S. Collins, NIH director, in a statement. “The hope is that this process may identify some potential new treatments for rare and neglected diseases.”

The research is being coordinated by the NIH’s Chemical Genomics Center and uses information on 27,000 active drug ingredients included in the center’s publicly available pharmaceutical collection browser. It also includes 2,750 small molecule drugs with regulatory approval in the United States, Canada, Europe and Japan as well as those registered for human clinical trials. The center hopes to add more compounds.

For now, the focus is on collaboration with disease foundations, industry and academic investigators who can test the limited amounts of the compounds in the database. New clinical trials would be needed to test the drugs on the rare, and neglected, diseases. As would U.S. Food and Drug Administration approval.

The cost of drug development is so high that there are only therapies for less than 300 rare diseases now. But the hope is now that some of these other drugs have been vetted in large populations that there uses can be expanded. There are a few cases of new uses being found for drugs already



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