Parent Project Muscular Dystrophy (PPMD announced that PPMD will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio, to conduct clinical testing of a promising gene therapy technique for muscle disease.
Investigators, led by Jerry Mendell, MD, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital with co-investigator Brian Kaspar, PhD, will inject a modified virus — vector — carrying the gene for the muscle growth-stimulating protein follistatin into the quadriceps muscles of volunteers with Becker muscular dystrophy and sporadic inclusion body myositis.
The goal of the study is to verify that the procedure is safe and to document any increase in quadriceps muscle size and function. People with these diseases have overall muscle weakness but with particular weakness of the quadriceps muscle, which is important for standing and sitting.
PPMD funding for the project will cover the costs of manufacturing the clinical grade viral vectors, and the costs associated with the clinical testing. If the initial study is successful, the investigators will expand the research to a phase II study and also will make plans to test it in Duchenne muscular dystrophy and other muscle diseases. The first clinical studies are planned for early 2011.
“This is the first time a gene therapy approach has been used to supply genes that generically stimulate muscle growth rather than directly replacing missing muscle proteins,” Sharon Hesterlee, PhD, PPMD senior director of research and advocacy, stated in a press release. “Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse.”
PPMD’s chief executive officer and president, Patricia Furlong, stated that she believes the work being done at Nationwide Children’s Hospital has huge potential.
“Part of PPMD’s mission has always been to fund promising research in the fight to end Duchenne,” she stated. “Not only does the work Dr. Mendell is doing with follistatin show great promise, but if successful, it would help all patients living with Duchenne and Becker muscular dystrophy, as well as other muscular issues. It complements the other exciting therapeutic avenues that PPMD is funding as well.”
Mendell acknowledged PPMD for the grant.
“PPMD is dedicated to supporting research and researchers that are navigating new paths in the treatment of muscular dystrophy. Pat and the PPMD community have not just been supportive financially, but have been true partners in this project,” he stated. “It is refreshing to work with an organization that is willing to take risks on treatments like follistatin. We look forward to continuing this partnership and sharing our results with the community.”