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Elizabeth Cooney is a health reporter for the Worcester Telegram & Gazette.
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Monday, January 29, 2007

'We wondered who was responsible for curing' ALS

Saying they can't wait for traditional research to bear fruit, two groups fighting amyotrophic lateral sclerosis will on Thursday toast their joint $36 million drug-discovery venture in Cambridge, a project led by people with personal ties to the disease.

The ALS Therapy Development Institute will have its $6 million annual budget matched by the Muscular Dystrophy Association for three years. The institute, founded in 1999, employs 35 people in Kendall Square but plans to hire 10 to 15 more, said institute president Sean A. Scott.

"Families banded together," said Scott, whose mother died of ALS. "Each of us had a relative with the disease. We wondered who was responsible for curing the disease. At the end of the day, nobody."

For the last five years the institute has screened FDA-approved drugs, looking for possible treatments for ALS. It will continue those tests while also searching for new compounds that might make it to Phase 1 clinical trials, CEO James Heywood said.

Better known as Lou Gehrig's disease, ALS is a chronic, progressive neurodegenerative disease that ends in paralysis. There are about 30,000 people with ALS in the United States.

Heywood's brother Stephen of Newton, who died in November, was the subject of a documentary film called "So Much So Fast."

Jamie Heywood was frustrated by the pace of ALS research.

"If we had decided to go to the moon the way we fund biomedical research, we'd have given $50,000 to 100 people and and wondered why we never got off the sidewalk," he said. "I think there's a need for programs that are rigorous and focused."

The institute describes itself as a "nonprofit biotech company" because of its industry-like approach to finding drugs that combat ALS or developing new ones. It appointed former Biogen Idec executive Steve Perrin as its chief scientific officer. Chairman Augie Nieto, for whom the Muscular Dystrophy Association's Augie's Quest is named, has ALS.

"There's a personal connection," Scott said. "The standards and aggressiveness are completely different when the next person taking the drug is a relative. It's not enough to have something published. It's got to work."

Posted by Elizabeth Cooney at 10:02 AM
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