ANTI-SEIZURE DRUG FAILS TO HELP IN SPINAL MUSCULAR ATROPHY

Muscular Dystrophy Association Following Other Leads

TUCSON, Ariz., Oct. 17, 2000 -- A clinical trial of the drug gabapentin for spinal muscular atrophy shows the drug failed to help people with this disorder, the Muscular Dystrophy Association announced today. The results were announced at a meeting of the American Neurological Association in Boston.

"I'm disappointed this drug didn't show any benefit for people with spinal muscular atrophy," said neurologist Leon I. Charash, chairman of MDA's Medical Advisory Committee. "But I'm encouraged by other recent research findings in SMA, and I think we can expect better medications in the future. At this point, it really doesn't make sense for doctors to prescribe gabapentin for SMA patients."

SMA is a genetic disorder involving the malfunction of muscle-controlling nerve cells (motor neurons) in the spinal cord. The disease leads to varying degrees of weakness, including loss of strength in vital respiratory muscles in its more severe forms. Some 30,000 people in the United States have SMA, which is among the neuromuscular disorders in MDA's program.

In its most severe form, known as SMA type 1, or Werdnig-Hoffmann disease, children usually don't survive more than about two years. SMA types 2 and 3 allow for longer survival, albeit with moderate to severe disability. The gabapentin trial studied people with types 2 and 3.

Gabapentin (marketed by Pfizer as Neurontin) interferes with the actions of the central nervous system chemical glutamate. The drug is primarily used for control of seizures, and it has FDA approval for this use. Excess glutamate is believed to be involved in motor neuron disorders such as Lou Gehrig's disease.

The SMA trial showed no benefit, based on measures of change in arm strength, breathing muscle function or overall function, said Robert Miller, an MDA-supported neurologist at California Pacific Medical Center in San Francisco, who was on the study team.

MDA continues to support extensive research in SMA. Recent research shows that increasing the amount of a protein known as SMN, crucial to motor neurons, may one day be a possible SMA treatment.

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research.  The Association also provides comprehensive health care and support services, advocacy and education. For more information, call (800) 572-1717 or visit www.mda.org.