New institute will study rare diseases

By Maggie Fox, Health and Science Editor

WASHINGTON (Reuters) - A unique new institute will look for ways to treat rare and neglected diseases and take the first and riskiest steps toward bringing new drugs to market, U.S. health officials said on Wednesday.

Congress has provided $24 million a year for five years to start the Therapeutics for Rare and Neglected Diseases Program, or TRND at the National Institutes of Health, acting NIH director Dr. Raynard Kington told reporters in a telephone briefing.

The program will use taxpayer money to get drugs through the most costly and dangerous phase of development, known as the "Valley of Death" because so many fail there.

It will publish details of failures as well as successes to guide other researchers, the NIH said.

"Twenty-five to 30 million Americans suffer from rare or neglected diseases," Kington said.

A rare disease is one that affects fewer than 200,000 Americans, and NIH estimates there are about 6,800 of these conditions, ranging from multiple symmetric lipomatosis or Madelung's disease, characterized by large fat deposits around the neck and nervous system abnormalities, to pseudomyxoma peritonei, in which tumor cells swell up the abdomen.

Only about 200 of these conditions, many of which affect fewer than a dozen people, have treatments.

"We don't know yet exactly which diseases this program will take on," Dr. Alan Guttmacher, acting director of the National Human Genome Research Institute, told the briefing.