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Parent Project Muscular Dystrophy Awards $600K to Nationwide Children's Hospital for Gene Therapy Study

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Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), 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, M.D., director of the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital with co-investigator Brian Kaspar, Ph.D., 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. Preliminary studies in mice with muscular dystrophy and in non-human primates demonstrated that follistatin delivered in this manner can cause significant increases in the size of injected muscles. Improvements in the strength of the mice and non-human primates were documented.

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 will also make plans to test it in Duchenne muscular dystrophy and other muscle diseases. The first clinical studies are planned to start in 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, " explains Sharon Hesterlee, Ph.D., PPMD Senior Director of Research and Advocacy. "Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse."

PPMD's CEO and President, Patricia Furlong 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. 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."

This grant was made possible in large part due to the generosity of Chris and Anna Stansbury and Robert H. Book.