These grants add to the $168 million already donated to ALS research by the MDA, including $20 million in just the past 10 years.
Apart from the federal government, the MDA is the largest source of research funding into diseases such as muscular dystrophy, ALS, and other neuromuscular disorders, committing more than $1 billion to stimulate the discovery of therapies and cures.
“We are at a time of unprecedented progress in ALS, thanks in large part to the foundation established by MDA’s investments,” Sharon Hesterlee, PhD, chief research officer of the MDA, said in a press release.
“We now have greater insight into the processes that underlie this disease and the ability to target specific genes where genetic causes are identified,” Hesterlee said.
Funds will be used to support the development of the first platform trial for ALS, enabling researchers at ALS clinics and MDA Care Centers to conduct clinical trials testing more than one experimental therapy at a time, as well as new biomarkers of ALS.
Paganoni’s team will promote clinical trial recruitment and maximize patient access by working with ALS centers, including 41 MDA Care Centers, and MDA’s MOVR data hub — a large database that contains genetic and clinical data from patients with neuromuscular disorders who have been followed at MDA Care Centers.
A $917,261 grant was awarded to Michael Benatar, MD, PhD, professor of Neurology at Miller School of Medicine of the University of Miami in Florida. Also given over three years, funds will support the launch of the ALS Toolkit — a software module incorporated on the Epic electronic health record (EHR) that will help collect data for research and quality-of-care improvements.
The ALS toolkit’s goal is to provide a standardized data collection platform across clinical sites nationwide. This may eliminate, or at least reduce, the need for patients to make separate visits for care or during clinical trials, which contributes to lower time demands for their physicians and themselves.
A one-year research grant totaling $137,228 also has been given to Jonathan Glass, MD, PhD, professor at Emory University in Atlanta, Georgia, to continue providing data and tissue samples from ALS patients and people without the disease who participated in Emory’s Clinical Research in ALS (CRiALS) project.
Glass’ team is currently managing a database containing long-term clinical data on ALS patients, as well as demographic information from people without the disease (control subjects). Tissue specimens from these participants are banked and matched according to demographic and clinical data. The project seeks to determine if failures in clinical trials of ALS may be due to unknown disease sub-types that do not respond to the therapy being tested.
These funds will be used by Philip C. Wong, PhD, investigator and professor of Pathology at Johns Hopkins, to develop a new gene therapy delivery system based on a harmless adeno-associated virus (AAV) to treat patients with ALS and frontotemporal degeneration (FTD).
Using this strategy, Wong hopes to create a new form of gene therapy that can be used to counteract the effects of mutations in the TARDBP gene, which have been found to cause FTD, as well as some forms of ALS.
Hiroshi Mitsumoto, MD, director of the ALS/PLS Center at Columbia University Medical Center in New York, received a one-year MDA research grant of $54,784 to support three studies funded by the National Institutes for Health (NIH) and Centers for Disease Control and Prevention (CDC).
Funds will back up statistical analyses aiming to discover new risk factors for ALS, as well as a potential cause for the disease. They also will be used to support a clinical trial that will assess the effects of a Japanese herbal medicine on muscle cramps, one of the most debilitating symptoms of ALS.
A full list of individual awards may be accessed at the MDA’s Grants at a Glance.
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