Nationwide Children’s Hospital Awarded $600,000 for Gene Therapy Clinical Trials

Parent Project Muscular Dystrophy (PPMD) has awarded Nationwide Children’s Hospital $600,000 to conduct clinical testing of a promising gene therapy technique for muscle disease.  PPMD is the largest non-profit organization in the US and is focused on finding a cure for Duchenne muscular dystrophy, the most common form of childhood muscular dystrophy. 

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.  

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.

For more on this breakthrough therapy, see the release from Nationwide Children’s Hospital

In a related story, Nationwide Children’s Hospital received an award from the Cardinal Health Foundation for its achievements in reducing adverse drug events.  The award was one of 40 awards given out by Cardinal in 2010 in recognition of efforts by US hospitals, health systems and community health clinics to improve efficiency and quality of care. 

For more on this story, see the release from Cardinal