ALBUTEROL SHOWS PROMISE IN PILOT STUDY
OF DMD, BMD

An MDA-supported pilot study of the drug albuterol in Duchenne muscular dystrophy (DMD) and its less severe variant, Becker MD (BMD), conducted at the University of California at Los Angeles, has shown encouraging results.

Albuterol, a drug that belongs to a class known as “beta-2 agonists” and is generally used to treat asthma, has long been thought to have positive effects on muscle. Evidence suggests it may increase muscle protein synthesis and slow muscle protein breakdown.

MDA research grantee Melissa Spencer, a muscle biologist at UCLA, with neurologist Michael Graves, who co-directs the MDA clinic at that institution, published the albuterol results in the March 23 issue of Neurology.

Nine boys 5 to 9 years old with DMD or BMD, all of whom were still walking, took part in the trial.

After baseline measurements of muscle strength and function, the boys were given 12 weeks of extended-release, oral albuterol (Proventil Repitabs), or a placebo. Neither the children nor the investigators knew which substance each boy was taking.

After a four-week break from medication or placebo, the boys switched groups, so that all participants received albuterol for either the first or second half of the trial.

When the researchers analyzed the results, they found that those on the drug showed better muscle strength than those on the placebo, mostly in the thigh muscles. Not all muscles showed increased muscle strength, and the researchers say they think some muscles may take up albuterol better than others.

Functional tests, such as the time for a boy to walk or run 30 feet, climb four steps, or rise from a back-lying to a standing position, were not improved by albuterol in this study. The researchers say it might require a longer albuterol treatment time to see an improvement in these functional measures as opposed to simple strength tests.

No cardiac abnormalities (which had been anticipated) or other side effects were noted.

A larger study, already under way with MDA support, will be necessary to confirm and extend these promising early results and reassure doctors and families that the drug is safe as well as effective in treating DMD and BMD.

For information about the larger study, please see the clinical trial posted on this site at http://www.mda.org/research/ct-albudmd.aspx.