WSJ Has Article On New NIH Drug Development Program

http://online.wsj.com/article/SB10001424052970203771904574177701770654102.html#articleTabs%3Darticle


The Wall Street Journal
MAY 21, 2009, 10:25 A.M. ET
NIH Launches Drug-Development Program for Rare Diseases

By JENNIFER CORBETT DOOREN
WASHINGTON -- The National Institutes of Health is wading into the drug-development process by launching a program aimed at finding and getting new drugs ready for testing in humans with rare diseases. The development program would also target so-called neglected diseases, while rare in the U.S., are often linked to parasites that sicken millions of people who live in tropical parts of the world. ”We’ve never tried to directly develop medicines for rare and neglected diseases,” said NIH Acting Director Raynard Kington, who announced the program Wednesday. NIH has traditionally engaged in basic science and has only had limited programs for developing drugs. The program comes with new money from Congress and is aimed at finding new treatments for some of the 6,800 rare diseases that collectively affect about 25 million Americans. Of those diseases only 200 have treatments, according to NIH.
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The project, known as Therapeutics for Rare and Neglected Diseases Program, will be overseen by the NIH Office of Rare Diseases Research. Stephen Groft, the director of the rare diseases office, said the program is starting with $24 million in funding this year with expectation of receiving the same amount each year until 2013, which effectively means the program would only be able to focus on a couple of compounds per year. ”Preclinical work is hard and our resources will be limited,” he said. However, he said other funds from NIH centers and existing programs can be used to augment the broader drug-development program. Groft said NIH would work with researchers and patient advocacy groups to identify possible molecules that could be developed into a potential treatment. The goal of the program is to turn over potential new medicines to private companies for further development. Information about chemicals that failed in tests will be published.
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I hope that ALSA, ALSTDI, MDA, Packard Center, Les Turner, etc. will speak to us on this and whether it can help ALS drug development.