NEW YORK, Sept. 30, 2020 /PRNewswire/ -- 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 over $168 million to ALS research, including $20 million in the last decade.
"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, chief research officer for MDA. "We now have greater insight into the processes that underlie this disease and the ability to target specific genes where genetic causes are identified."
The newly funded projects will aim to: create a clinical trials platform for testing multiple drugs simultaneously; deploy an ALS Toolkit; continue development of a biobank and database that can be used by ALS researchers .; 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 ALS symptoms, while also researching risk factors and the cause of ALS.
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 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. Dr. Paganoni's team will work with ALS centers (including 41 MDA Care Centers) and MDA's 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, a module built into the 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 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 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 from participants are banked and matched with clinical and demographic data. The project seeks to answer whether failures in ALS clinical trials are due to different types of ALS we are currently unaware of, that 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 hopes to compensate for the loss of normal RNA generation due to dysfunction related to TDP-43, a DNA binding protein that attaches itself to brain cells in some forms of ALS and the most common form of FTD..
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 three National Institutes for Health and Centers for Disease Control and Prevention-funded studies, for which MDA has already provided supplemental funding. The 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 ALS symptoms.
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, visit MDA's Grants at a Glance.
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 through innovations in science and care. As the largest source of neuromuscular disease research funding outside of the federal government, MDA has committed more than $1 billion 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 provides one-on-one specialized support, and we offer conferences, events, and materials for families and healthcare providers. During COVID-19, MDA continues to produce virtual events and programming to support our community. For more information, visit mda.org.