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

Tuesday, August 10, 2010 Hospital News
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Dr. Jerry Mendell Leading Follistatin Gene Therapy

MIDDLETOWN, Ohio, Aug. 9 /PRNewswire-USNewswire/ -- 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."

Dr. Mendell is extremely grateful to PPMD for the grant and acknowledges, "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. 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."

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

About Duchenne and Becker

Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function. Becker muscular dystrophy, which is less severe than Duchenne, occurs when dystrophin is manufactured, but not in the normal form or amount.

About PPMD

Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization's mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information, visit

SOURCE Parent Project Muscular Dystrophy

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