MDA PILOT STUDY FINDS FIRST DRUG TO PROVIDE HEALTH BENEFIT IN ADULT FORM OF MUSCULAR DYSTROPHY
Expanded Clinical Trial Begins
TUCSON, Ariz., May 20, 1998 -- The drug albuterol may increase muscle strength and bulk in at least one form of muscular dystrophy, say investigators who conducted a preliminary study with backing from the Muscular Dystrophy Association (MDA). This is the first medication to show promise as a therapeutic agent for an adult form of muscular dystrophy. An expanded trial of the drug in adults with facioscapulohumeral (FSH) muscular dystrophy is getting underway at the University of Rochester and Ohio State University.
"Quality of life therapeutics are vitally important to families living with neuromuscular diseases," said Dr. Leon I. Charash, Chairman of MDA's Medical Advisory Committee. "While rapid research progress gives us good reason to be hopeful for cures for FSH and other muscle-wasting disorders through gene therapy, the news that alubuterol can increase strength and overall muscle weight in a progressive disease is significant."
Albuterol belongs to a class of drugs known as beta receptor agonists and is marketed as a treatment for asthma and other lung conditions. It, or medications like it, could have beneficial effects in other forms of muscular dystrophy and in other muscle-wasting disorders, say the researchers. Albuterol isn't yet approved by the Food and Drug Administration for treatment of muscular dystrophy.
The pilot trial at two medical centers, results of which are published in the May issue of Neurology, tested albuterol in 15 adults with FSH muscular dystrophy. On average, strength increased by about 12 percent on standard measures, and overall muscle weight increased by about 3 pounds.
"This small preliminary study provided encouraging results and suggests that there may be a role for these kinds of agents in some types of muscular dystrophy and that they're worth studying in a larger trial," said neurologist and MDA research grantee John Kissel of Ohio State University Medical Center. Kissel coordinated the Ohio arm of the study.
He says that, although some doctors will probably now prescribe albuterol for muscular dystrophy based on the findings announced today, the research group doesn't yet recommend the drug because of the preliminary nature of the study and the small number of patients involved.
Neurologist Rabi Tawil at the University of Rochester (N.Y.) Medical Center coordinated the Rochester arm of the study. Kissel and Tawil are co-directors of MDA clinics at their institutions.
The study has been expanded at both centers. If you have FSH and would like information, call Lynn Cos at the University of Rochester at (716) 275-7680 or call Karen Downing or Cheryl Kacvinsky at Ohio State at (614) 292-1234.
FSH is one of nine forms of muscular dystrophy. It's genetic and generally begins in the early adult or teen years and starts with weakness in the face and shoulder muscles. Weakness slowly progresses to other muscles, and disability can become moderate to severe.
MDA researchers are studying a small area of chromosome 4 where the genetic defect for FSH is in most people with the disorder. Albuterol is the first treatment to show promise in FSH.
MDA is the nation's leading nongovernmental funder of research into neuromuscular diseases. By annually funding some 400 scientific teams worldwide, its investigators have made significant advances toward cures for several muscle-wasting disorders. They've also pioneered breakthroughs that may lead to therapies for heart disease, cancer, AIDS, Alzheimer's, Parkinson's, Huntington's, and cystic fibrosis.
MDA is the first voluntary health agency recognized by the American Medical Association with a Lifetime Achievement Award "for significant and lasting contributions to the health and welfare of humanity." The agency is working to defeat 40 neuromuscular diseases through programs of worldwide research, comprehensive patient and community services, and far-reaching public health education. For more information about MDA, and/or referrals to its clinics or its new ALS center, call 1-800-572-1717. Information about MDA is also available electronically through its Internet Web site (www.mda.org).
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