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Muscular Dystrophy Association Awards Five Grants Totaling Over $1.6 Million for Amyotrophic Lateral Sclerosis (ALS) Research

MDA continues to step up fight against ALS during this challenging time, building on its $168 million research commitment

New York, N.Y., Sept. 30, 2020 — The Muscular Dystrophy Association (MDA) announced today the awarding of five new MDA grants totaling over $1.6 million toward research focused on amyotrophic lateral sclerosis (ALS), commonly known as “Lou Gehrig’s disease.” MDA has devoted more than $168 million to ALS research, including $20 million in the last 10 years.

“We are at a time of unprecedented progress in ALS, thanks in large part to the foundation established by MDA’s investments,” says Sharon Hesterlee, PhD, executive vice president, chief research officer for Muscular Dystrophy Association. “We now have greater insight into the processes that underlie this disease, as well as the ability to target specific genes where genetic causes are identified. Thanks to our generous donors and the innovation of our grantees, we will have the ability to move even closer to a cure.”

The newly funded projects will aim to: help create a clinical trials platform for testing multiple drugs simultaneously; deploy an ALS Toolkit, which will enable MDA Care Centers serving ALS patients to better collect data for research and improved quality of care; continue development of a biobank and database that can be used by ALS researchers across the U.S.; develop and validate a new gene therapy strategy for ALS and frontotemporal degeneration (FTD), and prepare for a personalized clinical trial of a novel treatment for debilitating ALS symptoms, while also researching risk factors and the cause of ALS.

The ALS grant awards for this grant cycle include:

• Sabrina Paganoni, MD, PhD, co-director, MGH Neurological Clinical Research Institute of Massachusetts General Hospital, was awarded an MDA research grant totaling $500,000 over three years to continue development of the first Platform Trial for ALS. The project will enable ALS researchers at MDA Care Centers and other ALS clinics to conduct clinical trials of more than one drug at a time, as well as test novel indicators of ALS (biomarkers). Dr. Paganoni’s team will work with 54 ALS centers (including 41 MDA Care Centers) and MDA’s innovative MOVR data hub to promote clinical trial recruitment and maximize patient access.
• Michael Benatar, MD, PhD, professor of Neurology, Miller School of Medicine of the University of Miami, was awarded an MDA clinical research network grant totaling $917,261 over three years to support the multicenter deployment of the ALS Toolkit. The toolkit is a module built into the widely used Epic electronic health record (EHR) that will facilitate structured data collection for research and quality of care improvement. The toolkit will provide a platform for standardized collection of data across clinical sites nationwide, which may eliminate the need for patients to make separate visits for clinical trials and for care, and reduce time demands for both patients and physicians.
• Jonathan Glass, MD, PhD, professor, Department of Neurology and Pathology, Emory University, was awarded a one-year continued MDA research grant funding of $137,228 to continue to provide the ALS research community with data, biofluids, and tissues from deeply phenotyped ALS patients and people without ALS who participate in Emory’s Clinical Research in ALS (CRiALS) project. Deep phenotyping is a comprehensive analysis of phenotype abnormalities—clinically characterizing traits that signify health or disease—a process necessary for the development of precision medicines. Dr. Glass’ team maintains a database of long-term clinical data on ALS patients and demographic data on related and unrelated people who serve as research controls. Biospecimens such as blood, DNA and spinal fluid from participants are banked and matched with clinical and demographic data. The project seeks to answer the question: Are failures in ALS clinical trials due to the fact that there are different types of ALS we are currently unaware of, some of which may not respond to a specific drug being tested?
• Jeffrey Rothstein, MD, PhD, director of the Robert Packard Center for ALS Research at Johns Hopkins University School of Medicine, was awarded a one-year MDA research grant of $54,785 for investigator Philip C. Wong, PhD, professor of Pathology, to develop an adeno-associated virus (AAV) delivery method to validate a gene therapy strategy for ALS and frontotemporal degeneration. The proposed strategy is hoped to compensate for the loss of normal RNA generation due to dysfunction related to TDP-43. TDP-43 is a DNA binding protein that attaches itself to brain cells in some forms of ALS and the most common form of FTD, causing brain dysfunction and degeneration.
• Hiroshi Mitsumoto, MD, DSci, director of the ALS/PLS Center at Columbia University Medical Center, was awarded a one-year MDA research grant of $54,784 to support continuation of three National Institutes for Health and Centers for Disease Control and Prevention-funded studies, for which MDA has already provided supplemental funding. The new MDA grant will be used to conduct crucial statistical analyses to uncover ALS risk factors and a potential cause of ALS. The funding will also be used to prepare for a personalized clinical trial of a Japanese herbal medication, to determine if the medication can improve muscle cramps, one of the most debilitating symptoms experienced by people with ALS.

Due to COVID, MDA had to cancel its spring review session. The funding of these projects was reviewed in 2019. For a complete list of individual awards for 2020 and prior grant cycles, visit MDA’s website and explore the Grants at a Glance section.

Grants being given jointly by MDA and other organizations will be announced separately.

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.