By VCU News staff

Researchers at Virginia Commonwealth University and the University at Albany have been awarded a $6.5 million grant from the National Institutes of Health to establish a Wellstone Muscular Dystrophy Specialized Research Center.

Named in honor of the late U.S. Sen. Paul D. Wellstone, a champion of muscular dystrophy research, the Wellstone MDSRC program was established in 2003 by the NIH to support innovative research into the group of inherited genetic conditions, which are marked by progressive muscle weakness and loss. The new center will be one of just six Wellstone locations in the nation.

VCU and UAlbany will receive $6.5 million over five years to establish the center under the co-leadership of neurologist Nicholas Johnson, M.D., director of VCU's Center for Inherited Muscle Research, and biochemist Andy Berglund, Ph.D., director of UAlbany's RNA Institute and the university's Keith Hynes Endowed Professor in STEM.

The new VCU/UAlbany Wellstone center will leverage the universities' combined expertise in the leading form of adult-onset muscular dystrophy in the United States - myotonic dystrophy.

The NIH's P50 grant, which supports specialized centers, offers funding for multidisciplinary research focused on a specific disease or biomedical problem, and that integrates several interrelated research projects under a unified theme. The Wellstone P50 centers are supported by four NIH units: the National Institute of Arthritis and Musculoskeletal and Skin Diseases; the Eunice Kennedy Shriver National Institute of Child Health and Human Development; the National Heart, Lung and Blood Institute; and the National Institute of Neurological Disorders and Stroke.

The VCU/UAlbany Wellstone center, which will share expertise and resources with the Wellstone-funded and broader muscular dystrophy research communities, will focus primarily on two research projects that will address major challenges for myotonic dystrophy. The work will be led by principal investigators from both VCU and UAlbany.

Project 1, led by Johnson and VCU neurologist Sam Carrell, M.D., Ph.D., will develop clinical and biomarker outcomes to prepare the most affected population - children with myotonic dystrophy - for upcoming clinical trials.

Project 2, co-led by Berglund and Johnson, will identify genetic elements that make the disease highly variable and affect the outcome of therapeutics.

The VCU/UAlbany Wellstone center will also involve an:

• Administrative core to collaborate with patient advocacy groups and industry partners to support the center's work.
• Resource core to centralize access to research cell lines, samples, and genomic and phenotypic data for Wellstone investigators and the muscular dystrophy community.
• Training core to foster the development of the next generation of researchers and clinicians who will support future research and clinical muscular dystrophy programs.

These cores will involve VCU neurologist Melissa Hale, Ph.D., and administrator Erin DeSpain, as well as UAlbany's Berglund and John Cleary, Ph.D., the RNA Institute's assistant director of research.

"CIMR is at the forefront of moving VCU into an era where genetic therapies offer unprecedented hope for patients with devastating neuromuscular diseases," said P. Srirama Rao, Ph.D., VCU's vice president for research and innovation. "The fundamental work by their talented faculty and staff, and their ability to conduct both transdisciplinary and team science research, is crucial in gathering critical data and patient outcomes to create a future where the debilitating effects of myotonic dystrophy can be mitigated."

"The RNA Institute is an exemplar of the University at Albany's commitment to research that serves the public good," said university President Havidán Rodríguez, Ph.D. "UAlbany researchers are leveraging the power of collaborations with other leading universities, like VCU, to conduct trailblazing science that improves lives. We can't solve medicine's most daunting challenges alone, but through impactful collaborations like this one with VCU, Dr. Berglund and his colleagues at the RNA Institute are advancing the development of cutting-edge diagnostics, technologies and therapeutics. I know this important work is a source of hope to families impacted by this terrible disease."

"Our mission is to bring hope to patients and families affected by myotonic dystrophy and other muscular dystrophies," Johnson and Berglund said. "Through collaboration, innovative research and a patient-centered clinical approach, this Wellstone center will help transform the future of neuromuscular research and care."