NIH announces new program to develop therapeutics for rare and neglecte...( The National Institutes of Health is...)

The National Institutes of Health is launching the first integrated, drug development pipeline to produce new treatments for rare and neglected diseases. The $24 million program jumpstarts a trans-NIH initiative called the Therapeutics for Rare and Neglected Diseases Program, or TRND.

The program, funded by Congress this spring, is unusual because TRND creates a drug development pipeline within the NIH and is specifically intended to stimulate research collaborations with academic scientists working on these illnesses. The NIH Office of Rare Diseases Research will oversee the program, and TRND's laboratory operations will be administered by the National Human Genome Research Institute (NHGRI), which also operates the NIH Chemical Genomics Center, a principal collaborator in TRND. Other NIH components will also participate in the initiative.

A rare disease is one that affects fewer than 200,000 Americans. NIH estimates that more than 6,800 rare diseases afflict more than 25 million Americans. However, effective pharmacologic treatments exist for only about 200 of these illnesses. Many neglected diseases also lack treatments. Unlike rare diseases, however, neglected diseases may be quite common in some parts of the world, especially in developing countries where people cannot afford expensive treatments. Private companies seldom pursue new therapies for these types of illness because of high costs and failure rates and the low likelihood of recovering investments or making a profit.

"NIH is gratified that Congress supports finding solutions for millions of our fellow citizens faced with rare or neglected illnesses," said NIH Acting Director Raynard S. Kington, M.D., Ph.D. "The federal government may be the only institution that can take the financial risks needed to jumpstart the development of treatments for these diseases, and NIH clearly has the scientific capability to do the work. We are eager to begin."

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