Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research
Critical funding provided by MDA during this challenging time will support studies to better understand and address disease mechanisms, build on existing gene therapies and other treatments, advance drug target identification and stop disease progression
New York, N.Y., Sept. 23, 2020 — The Muscular Dystrophy Association (MDA) announced today the awarding of 15 new MDA grants totaling more than $4 million toward research focused on a variety of neuromuscular diseases (NMDs), including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), Becker’s muscular dystrophy (BMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). This round of grant funding reinforces MDA’s unwavering commitment—in the face of declining income due to the COVID-19 pandemic—to the progress of neuromuscular disease research and builds on the more than $1 billion MDA has already invested in research to uncover new treatments and cures for NMDs since its inception. Some grants will go into effect this year, while others will be awarded in 2021.
“We continue to fund the most innovative research that will lead us to cures for a range of neuromuscular diseases,” says Sharon Hesterlee, PhD, executive vice president and chief research officer for Muscular Dystrophy Association. “We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable.”
Dr. Hesterlee added, “Although COVID led the cancellation of MDA’s spring review session, we are pleased to announce the funding of these projects, which were reviewed in 2019.”
The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas. The awarded grants will fund studies to further advance our understanding of genetic causes of and risk factors for NMDs, investigate new approaches to developing gene therapies and other innovative potential treatments, including stopping disease progression and improving genetic testing technologies.
- Jeffrey Chamberlain, PhD, McCaw Endowed Chair in Muscular Dystrophy, University of Washington, was awarded an MDA research grant totaling $471,694 over two years to develop improved ways to deliver gene therapy in Duchenne muscular dystrophy. Two of the gene-therapy delivering vectors developed by the Chamberlain lab are currently being tested in clinical trials. If successful, the development of these new vectors may significantly improve health outcomes for children receiving gene therapy for DMD.
- Qi Lu, MD, PhD, director of the McColl Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, Charlotte, NC, has been awarded an MDA research grant totaling $350,053 over two years to develop a way to reduce the death of heart muscle cells known as cardiomyocytes and improve their capacity to regenerate. Heart failure is a common cause of death in people with all types of muscular dystrophy, due to accumulation of fibrosis that leads to poor regenerative capacity in the heart muscle, and ultimately heart failure. Dr. Lu’s research may provide new hope for all heart failure patients.
- Stephan Zuchner, MD, PhD, professor and chair, Dr. John T. MacDonald Department of Human Genetics, University of Miami, will be awarded an MDA research infrastructure grant totaling $163,920 over three years to expand and make more widely available resources to streamline gene identification and therapy development for Charcot-Marie-Tooth (CMT) disease, using a genomic data infrastructure platform developed by Dr. Zuchner. About 40% of people with CMT do not have a confirmed genetic diagnosis. Making genetic data available to researchers around the world may improve diagnostic processes, guide functional studies and drug discovery and build the foundation for a patient selection process that is urgently needed for well-designed CMT clinical trials.
- Charles Thornton, MD, Saunders Family Distinguished Professor in Neuromuscular Research, University of Rochester Medical Center, was awarded an MDA research grant of $49,953 for one year to make improvements in technology for genetic testing of myotonic dystrophy type 1, a relatively common form of muscular dystrophy. Many people with DM1 have never had genetic testing to determine the size of the expansion of DNA building blocks known as CTG, which repeat in the non-coding region of the DM protein kinase gene—the cause of this type of muscular dystrophy. Dr. Thornton and his team have the goal of developing methods of genetic analysis of DM1 that are as cost-effective and precise as possible.
- Erik Henricson, PhD, assistant professor, Physical Medicine and Rehabilitation, University of California, Davis, was awarded an MDA neuromuscular disease clinical research grant totaling $427,822 over three years to develop new and innovative mobile device-compatible methodologies with the goal of providing cost-efficient tools to allow consumers, clinicians and researchers to better evaluate the community mobility of people with neuromuscular diseases in clinical and research settings. These new tools will be based on low-cost and easily obtained accelerometers and machine learning algorithm technologies. The successful completion of this project will help assess the mobility of people living in the community who have mobility impairments due to neuromuscular diseases and other causes.
ALS grants will be announced separately later this month, as will grants being given jointly by MDA and other organizations.
About the Muscular Dystrophy Association
For 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this through innovations in science and innovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures. Research we have supported is directly linked to life-changing therapies across multiple neuromuscular diseases. MDA's MOVR is the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports the largest network of multidisciplinary clinics providing best in class care at more than 150 of the nation's top medical institutions. Our Resource Center serves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence at summer camp and through recreational programs, at no cost to families. During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA’s COVID-19 guidelines and virtual events are posted at mda.org/COVID19. For more information, visit mda.org.
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