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Drug fails in US study to help people with ALS
Mon Nov 24, 2008 4:02pm EST
By Will Dunham

WASHINGTON, Nov 24 (Reuters) - A two-year study of a drug that previously showed some potential for helping people with amyotrophic lateral sclerosis, or Lou Gehrig's disease, found that it gave no benefit at all, researchers said on Monday.

There is currently only one drug available for people with ALS, an incurable and fatal disease that affects nerve cells in the brain and the spinal cord, and it provides only the modest benefit of extending survival by perhaps a few months.

Scientists are eager to find something better. The new study was designed to be the definitive test of the Cephalon Inc (CEPH.O: Quote, Profile, Research, Stock Buzz) experimental insulin-like growth factor-1, or IGF-1, drug also known as Myotrophin.

The research involved 210 men and 120 women with ALS at 20 medical centers throughout the United States, with half getting injections twice a day of this growth hormone and half getting similar injections of a placebo.

After two years, those who got the drug did no better in terms of muscle strength, daily functioning or survival, Dr. Eric Sorenson of the Mayo Clinic in Rochester, Minnesota, and colleagues wrote in the journal Neurology.

"We found that the drug doesn't work," Sorenson said.

Cephalon, based in Frazer, Pennsylvania, donated the drug for use in the study but had no control over how the research was conducted, Sorenson said.

"We have no plans for further studies of Myotrophin and no other medications in development for ALS," Cephalon spokeswoman Sheryl Williams said by e-mail.

"Many companies have tried to develop medications for ALS but after more than a decade there is still only one FDA-approved medication for these patients," Williams said.

Two previous studies had looked at whether the drug can treat ALS, with one seeming to indicate the growth hormone might offer promise while the other finding no value at all. This third study was intended to settle the matter.

"I think it was important to resolve whether there was potentially some benefit. I think the disappointment is, of course, that we are desperate in ALS for a treatment for the disease," Lucie Bruijn of the ALS Association, which helped fund the study, said in a telephone interview.

ALS, one of most common neuromuscular diseases worldwide, causes muscles to weaken and eventually deteriorate. It usually develops in people between the ages of 40 and 60 and gets progressively worse over time.

It is sometimes called Lou Gehrig's disease after U.S. baseball legend Lou Gehrig of the New York Yankees, who died of ALS in 1941. Half of those diagnosed with ALS die within three years and 80 percent die within five.

"This disease's prognosis is as bad as any disease in medicine. The fact that we still have little to offer these people is so disappointing," Sorenson said in a telephone interview.

Sanofi-Aventis's (SASY.PA: Quote, Profile, Research, Stock Buzz) riluzole, or Rilutek, is the only medication approved by the U.S. Food and Drug Administration for ALS and it seems to slow the disease's progression and prolong life by a few months in some people.

The FDA previously declined to approve the use of Myotrophin for ALS.

An estimated 5,600 people are diagnosed with ALS each year in the United States with up to 30,000 people living with the disease at any given time, according to the ALS Association.

http://www.reuters.com/article/marke...52067620081124