CHoR neuromuscular expert will co-lead research efforts in the center aimed at tackling muscular dystrophy

Researchers at VCU and the University at Albany have been awarded a $6.5 million P50 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 National Institutes of Health to support innovative research into muscular dystrophies. The new center will be one of just six Wellstone awards in the nation.

Leveraging expertise in myotonic dystrophy

With this award, VCU and UAlbany will receive $6.5 million over five years to establish the center under the leadership of Dr. Nicholas Johnson, director of VCU’s Center for Inherited Muscle Research and a provider for pediatric and adult patients, and UAlbany’s Keith Hynes Endowed Professor in STEM Andy Berglund, director of UAlbany’s RNA Institute.

Johnson and Berglund will co-direct the Wellstone center, which will leverage the universities’ combined expertise in the leading cause of adult-onset muscular dystrophy in the United States – myotonic dystrophy.

P50 Center grants from the NIH provide funding for multidisciplinary research focused on a specific disease or biomedical problem that integrates several interrelated research projects under a unified theme. The Wellstone P50 centers are supported by 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 will share expertise and resources with the Wellstone-funded and broader muscular dystrophy research communities and 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’s Dr. Sam Carrell, 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 center will also involve an:

• Administrative core to collaborate with DM patient-advocacy groups and industry partners to support the center’s work;
• Resource core to centralize access to research cell lines, samples, genomic, and phenotypic data for Wellstone investigators and the muscular dystrophy community; and
• 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’s Melissa Hale and Erin DeSpain, and UAlbany’s Berglund and John Cleary, the RNA Institute’s assistant director for research.

Unprecedented hope for people with neuromuscular disease

“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., vice president for research and innovation at Virginia Commonwealth University. "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. UAlbany researchers are leveraging the power of collaborations with other leading universities, like VCU, to conduct trailblazing science that improves lives,” said University at Albany President Havidán Rodríguez. “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” said Johnson and Berglund. “Through collaboration, innovative research, and a patient-centered clinical approach, this Wellstone Center will help transform the future of neuromuscular research and care.”

Learn more about comprehensive care for kids with neuromuscular conditions at CHoR.