Genome.gov | 2011 Release: NIH researchers create comprehensive collection of approved drugs to identify new therapies for rare and neglected diseases

"Researchers have begun screening the first definitive collection of thousands of approved drugs for clinical use against rare and neglected diseases. They are hunting for additional uses of the drugs, hoping to find off-label therapies for some of the 6,000 rare diseases that afflict 25 million Americans. The effort is coordinated by the National Institutes of Health's Chemical Genomics Center (NCGC).

"This is a critical step to explore the full potential of these drugs for new applications," said NIH Director Francis S. Collins, M.D., Ph.D. "The hope is that this process may identify some potential new treatments for rare and neglected diseases."

The researchers assembled the collection of approved drugs for screening based on information from the NCGC Pharmaceutical Collection browser at http://tripod.nih.gov/npc. This publicly available, Web-based application is described in a paper appearing in the April 27 issue of Science Translational Medicine. It provides complete information on the nearly 27,000 active pharmaceutical ingredients, including 2,750 small molecule drugs 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. ....cont'd