University of Florida Health has been designated by the National Institutes of Health as one of six centers to help translate scientific findings into strategies to improve the lives of muscular dystrophy patients. UF Health has received first-year funding of a 5-year, $10.76 million grant to study ways to address muscle degeneration caused by the disease.
The grant was awarded to H. Lee Sweeney, Ph.D., director of the UF Myology Institute and the Thomas H. Maren, M.D., eminent scholar chair in pharmacology and therapeutics in the UF College of Medicine. He will serve as director of the newly designated Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at UF Health.
Muscular dystrophy is a genetic disease that causes muscle weakness and muscle loss, and can lead to muscle being replaced by fat or scar tissue. Some forms of the disease affect the heart muscle.
“We are very fortunate to have UF Health designated as a Wellstone Muscular Dystrophy Cooperative Research Center,” said David S. Guzick, M.D., Ph.D., senior vice president for health affairs at UF and president of UF Health. “The leadership and knowledge provided by Dr. Sweeney and his research colleagues will allow our Center to pursue scientific discoveries to improve muscular dystrophy therapies for people throughout Florida and around the world.”
The UF Health Wellstone Center’s main goal is to conduct translational research that will bring promising treatments into clinical trials for muscular dystrophy patients, Sweeney said. Specifically, the center’s researchers will work on better understanding and counteracting the way muscular dystrophies cause inflammation and scarring. By addressing those issues, they hope to improve the body’s ability to repair skeletal muscles and preserve cardiac function.
Researchers also will conduct collaborative studies to learn more about the mechanisms of muscular dystrophy that cause muscle scarring and the accumulation of fat issue. Existing and investigational drugs will be tested in mouse models on Duchenne muscular dystrophy, the most common form of the disease in children. That variation of the disease affects one out of every 3,600 male infants and typically reduces life expectancy to about age 30.
“The establishment of a Wellstone Center at UF and Dr. Sweeney’s abilities as a research pioneer puts us at the forefront of crucial research targeting muscular dystrophy,” said Michael L. Good, M.D., dean of the UF College of Medicine.
Having a Wellstone Center at UF Health will result in more clinical trials being based in Gainesville, according to Sweeney. Additional staff and postdoctoral associates also will be hired.
“This is an important step for realizing the University of Florida’s goal of being one of the preeminent centers for muscle disease research in the world,” Sweeney said.
The Wellstone Center brings together researchers from three UF colleges and five departments as well two other universities, said Glenn A. Walter, Ph.D., a professor in the College of Medicine’s department of physiology and functional genomics. Walter is part of a group doing an ongoing Wellstone project that uses magnetic resonance imaging and spectroscopy to monitor the functional changes caused by muscular dystrophy.
One unique aspect of UF’s Wellstone Center will be a comprehensive approach that includes a detailed study of the molecular and cellular origins of muscle degeneration coupled with innovative magnetic resonance imaging techniques developed as part of the National High Magnetic Field Laboratory, Walter said. The Magnetic Field Laboratory, which has facilities at UF, Florida State University and Los Alamos National Laboratory, is the world’s largest and highest powered magnet lab.
“The Wellstone Center will also allow for the far-reaching training of new scientists and clinicians in muscular dystrophy research and care all the way from the undergraduate level to practicing clinicians,” Walter said.
Another Wellstone investigator, Krista Vandenborne, P.T., Ph.D., professor and chair of the department of physical therapy in the College of Public Health and Health Professions, said being one of six Wellstone Centers allows scientific knowledge to flow more quickly and also expedites collaboration and idea sharing. She wants to further study the imaging of respiratory muscles affected by muscular dystrophy and expand the study of cardiac muscles. It’s important to understand the disease mechanics that cause muscular disorders to advance rapidly in some patients, she said.
“It really puts UF firmly on the map in terms of being one of the world leaders in neuromuscular disease research,” she said.
In addition to Sweeney, Vandenborne and Walter, UF Health investigators already doing Wellstone work include Elizabeth R. Barton, Ph.D., a professor in the department of applied physiology and kinesiology; Barry J. Byrne, M.D., Ph.D., director of the UF Powell Gene Therapy Center and a professor in the College of Medicine’s departments of pediatrics and molecular genetics and microbiology; and Andrew Judge, Ph.D., an associate professor in the department of physical therapy, who will lead the training and educational component.
The grant and the Wellstone Center designation are significant because learning more about the mechanisms that cause muscle diseases could generate more interest in clinical trials among large pharmaceutical companies, Byrne said. It’s also an acknowledgement that UF Health has developed significant expertise and potential in basic science and translational research, which aims to produce new drugs and treatments for patients, he added.
UF Health’s Wellstone Center is funded by the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases under award number U54AR052646. NIAMS has supported this project since 2005. The Wellstone Centers, which are located at universities and medical research facilities, work individually and collaboratively on basic and clinical research projects. They also make their core resources available to the national muscular dystrophy research community.