Steroid helpful in muscular dystrophy

Science News, August 22, 1987

Steroid helpful in muscular dystrophy

Using the steroid prednisone in patients with Duchenne muscular dystrophy (DMD) may delay by an average of two years their confinement to a wheelchair, say scientists at the Johns Hopkins University School of Medicine in Baltimore. Daniel B. Drachman and his coauthors report in the August ARCHIVES OF NEUROLOGY that the 16 prednisone-treated patients studied were able to walk and stand until an average age of 12, whereas the 38 untreated subjects lost their mobility by an average age of 10.

A genetic disease that affects young boys, DMD causes progressive weakening of the muscles, resulting in the need for a wheelchair by the age of 9 to 11 years. To assess the long-term effects of prednisone, the Baltimore study followed some patients as long as 14 years until they lost mobility. Among those patients who received prednisone for the longest period of time, loss of mobility was postponed more than three years compared to the control group. The treatment, however, does not stop the disease, and can produce side effects such as weight gain, hyperactivity and a rounded face.

Despite its limitations, researchers say that prednisone may prove itself as the first available treatment for the disease, which affects 1 in 3,000 live male births. In a six-month study also reported in ARCHIVES, 33 Duchenne patients treated with prednisone at four other medical centers showed definite improvement in muscle strength, although eventual immobility was not prevented. How prednisone acts to improve or slow the onset of symptoms is unclear. Authors of the reports suggest that prednisone's known anti-inflammatory capabilities may play a role, as may its ability to stabilize leaky muscle-cell membranes.