Sept. 16, 2010
UI's Wellstone Muscular Dystrophy Center awarded $7.8 million grant
The Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at the University of Iowa has received a five-year, $7.8 million grant renewal from the National Institute of Neurological Disorders and Stroke to advance its work on finding treatments for muscular dystrophies.
The UI center, which was established in 2005, brings together researchers and clinicians to translate laboratory discoveries into clinical applications to treat a group of congenital and limb girdle muscular dystrophies caused by abnormalities in a protein called dystroglycan.
Led by center director Kevin Campbell, Ph.D., professor and head of molecular physiology and biophysics at the UI Roy J. and Lucille A. Carver College of Medicine and a Howard Hughes Medical Institute investigator, the researchers are exploring ways to restore normal functioning dystroglycan to muscle cells to treat this group of muscular dystrophies, which includes Fukuyama Congenital Muscular Dystrophy, Walker-Warburg Syndrome, Muscle-Eye-Brain disease and limb-girdle muscular dystrophy 2I.
"Our strength lies in our interdisciplinary nature. We have basic and clinical scientists working and training together to find ways to help patients and families affected by these muscular dystrophies," said Campbell, who also is the Roy J. Carver Chair of Molecular Physiology and Biophysics and a professor of internal medicine and neurology. "In addition, our collaboration with other Wellstone Centers around the country is helping to create an infrastructure that will support clinical trials to test new therapies and diagnostics for these currently incurable conditions."
In addition to Campbell, the UI team is led by center co-director Steven Moore, M.D., Ph.D., UI professor of pathology, and Katherine Mathews, M.D., director of pediatric neurology with UI Children's Hospital.
Campbell will oversee a variety of pre-clinical studies using animal models and patient cells to test strategies for restoring normal dystroglycan function to muscle. Mathews will lead the clinical component of the project, which will include collecting data and creating an infrastructure to move possible treatments to clinical trials.
Moore and colleagues will coordinate a muscle-tissue/cell-culture/diagnostics core facility, which will serve as a national resource for research, as well as a laboratory for patient diagnostic testing and post-intervention biopsy evaluation for clinical trials.
The center will also focus on research training and education. A medical student fellowship will enable medical students to perform research and participate in the care of patients with these disorders. The center will also support a postdoctoral fellowship and a symposium to educate and engage patients and patient advocates.
"This training effort is vital," Campbell noted. "We need to ensure that there are future basic scientists and qualified clinicians who can work together effectively to develop and test potential treatments for these devastating conditions."
Muscular dystrophies are a diverse group of genetic diseases that cause progressive muscle wasting and weakness in affected individuals. The various types of muscular dystrophy differ in severity, age of onset, specific muscles affected and the rate at which symptoms progress. More than one million Americans have muscular dystrophy or a related neuromuscular disorder.
The UI center is one of six around the country named after Sen. Paul D. Wellstone (D-Minn.) who died in 2002. As a senator, Wellstone was instrumental in passing the Muscular Dystrophy Community Assistance, Research, and Education Amendments, which mandates that the NIH establish centers of excellence for basic and clinical research into Duchenne and other forms of muscular dystrophy.
To learn more about the UI Muscular Dystrophy Cooperative Research Center, visit http://www.medicine.uiowa.edu/mdcrc.
STORY SOURCE: University of Iowa Health Care Media Relations, 200 Hawkins Drive, Room W319 GH, Iowa City, Iowa 52242-1009