In a pre-clinical study, scientists from Wyeth Research and the University of Pennsylvania School of Medicine found that blocking the activity of a gene known as myostatin may reduce muscle degeneration. Myostatin is known to play a role in the growth and development of muscle. This new approach may provide a novel strategy for treatment of diseases associated with muscle degeneration, such as muscular dystrophy.

Using a mouse model that exhibits the muscle degenerative characteristics of Duchenne muscular dystrophy (DMD), scientists studied the effects of blocking myostatin. Mice were given weekly injections of a blocking antibody to myostatin over a three-month period. Compared to a control group of mice who did not receive the blocking antibody, treated mice had greater muscle size and strength. In addition, there was less evidence of muscle degeneration in the treated mice, suggesting that blocking myostatin slows the progression of this muscle disease in mice.

DMD is a hereditary degenerative disease of skeletal (voluntary) muscles and is considered the most prevalent form of childhood muscular dystrophy. The disorder typically is recognized from approximately age three to six years and has a relatively rapid, progressive disease course. DMD is initially characterized by muscle weakness and wasting (atrophy) within the pelvic area that may be followed by involvement of the shoulder muscles. With disease progression, muscle weakness and atrophy affect the trunk and forearms and gradually progress to involve most major muscles of the body.

"At present, there is no therapy offered for patients with DMD and we are encouraged by these pre-clinical results in mice," says Frank Walsh, Senior Vice President and Head of Discovery, Wyeth Research. "This compound might provide a way of altering the progression of DMD and other types of muscular dystrophy."

Other applications for this research may include new approaches to the treatment of Type II diabetes and frailty but research in these areas is also in very early stages.