$1 Million to Support Muscular Dystrophy Research
The School of Medicine is now one of only six Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers in the United States. The Center at Penn will be directed by Dr. H. Lee Sweeney, chair of the department of physiology; and co-directed by Dr. Kathryn R. Wagner, of The Johns Hopkins School of Medicine.
“This award will accelerate the pace of our translational work and provide a pathway to patient trials,” says Dr. Sweeney, in describing the work of the new Wellstone Muscular Dystrophy Cooperative Research Center at Penn. “It will also be the nucleus of a larger translational-research initiative for muscular dystrophies that I hope to catalyze at Penn.”
All six Centers, which honor the memory of the late-Senator Wellstone—who was a champion of muscular-dystrophy research and issues in Congress—are funded by the Muscular Dystrophy Association and the following three divisions within the NIH: the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), the National Institute of Neurological Disorders and Stroke (NINDS), and the National Institute of Child Health and Human Development (NICHD).
Under Dr. Sweeney’s leadership, the Penn-based Center will focus on ways to increase muscle growth and examine compounds to inhibit enzymes involved in the degradation of muscle tissue. The core facility, located at Penn, will analyze muscular dystrophy (MD) animal models. Planned clinical trials, to be based at NINDS in Bethesda, Maryland, will determine the safety and feasibility of a potential drug treatment for MD, which was first developed in Dr. Sweeney’s research lab. Other research sites, in addition to Johns Hopkins, that are contributing to the investigations directed by the Penn Center are the University of Florida, Gainesville, and the NINDS Intramural Research Program.
Muscular Dystrophy is characterized by progressive weakness and degeneration of the skeletal or voluntary muscles that control movement. Researchers at the Wellstone Centers study various forms of MD, including Duchenne/Becker Muscular Dystrophy, Myotonic Dystrophy, Facioscapulohumeral Dystrophy, and Limb-Girdle Muscular Dystrophy.
The two other most recently named Centers are the Children’s National Medical Center in Washington D.C. and at the University of Iowa. The three new centers join centers at the University of Washington, the University of Pittsburgh, and the University of Rochester. The centers work individually and collaboratively and are guided by a steering committee that includes representatives from each center. Each has both basic and clinical research projects and one or more core facilities to support them. Centers must also make core resources or services available to the national muscular dystrophy research community.
Almanac, Vol. 52, No. 16, December 20, 2005
December 20, 2005
Volume 52 Number 16