Research Shows Promise for Potential New Gene Therapy Strategy for Muscle-Wasting Diseases

March 10, 2008

Investigators in The Research Institute at Nationwide Childrens Hospital have identified the role of a protein that could potentially lead to new clinical treatments to combat musculoskeletal diseases, including Duchenne muscular dystrophy (DMD).

Results of these studies appear in the March 11, 2008 issue of the Proceedings of the National Academy of Sciences.

These studies, led by Brian Kaspar, PhD, a principal investigator in the Center for Gene Therapy at The Research Institute and an assistant professor of Pediatrics at The Ohio State University, focus on a protein called follistatin (FS). Using a single injection, gene-delivery strategy involving FS, investigators treated the hind leg muscles of mice. Results showed increased muscle size and strength, quadruple that of mice treated with proteins other than FS. The muscle enhancements were shown to be well-tolerated for more than two years.

According to Dr. Kaspar, increased muscle mass and strength were also evident when this strategy was tested using a model of DMD. Apart from the injected hind leg muscles, strengthening effects were also shown in the triceps. In addition, fibrosis, abnormal formation of scar tissue and a hallmark of muscular dystrophy, was decreased in FS-treated animals. 

We believe this new FS strategy may be more powerful than other strategies due to its additional effects, including its ability to reduce inflammation, said Dr. Kaspar.

The strategy showed no negative effects on the heart or reproductive ability of either males or females. The results were also replicated in older animals, suggesting that this strategy could be useful in developing clinical treatments for older DMD patients. 

This research provides evidence of multiple potential treatment applications for muscle diseases including, but not limited to, muscular dystrophy, said Jerry Mendell, MD, director of the Center for Gene Therapy at The Research Institute, a co-author on the study, and professor of Pediatrics in Neurology and Pathology at The Ohio State University. These results offer promise for treatment of potentially any muscle-wasting disease, including muscle weakness due to other illnesses, aging, and inflammatory diseases such as polymyositis. Our next step is to pursue clinical trials.

The Research Institute at Nationwide Childrens Hospital has a patent pending on the FS technique due to the major role it may play for muscular dystrophy treatment and other muscle-wasting diseases.

About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report’s 2017-18 list of “America’s Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric healthcare systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of nearly 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.