Muscular Dystrophy Association Awards $11 Million In New Research Grants

CHICAGO, Feb. 11, 2016 /PRNewswire-USNewswire/ -- After a historic unveiling of a revitalized rebranding in January, MDA today announced the award of 41 new research, development and research infrastructure grants totaling nearly $11 million and covering 13 different diseases. As part of its bold new plan to find treatments and cures for muscular dystrophy, ALS and related muscle-debilitating diseases, MDA announced it will double spending on research toward drug development and clinical trials by the year 2020 as part of its renewed mission focus.

"On the heels of unveiling our bold plan to find breakthroughs that will accelerate treatments and cures, this cycle's investment is a powerful proof point of our determination," said Valerie Cwik, M.D., MDA's Executive Vice President and Chief Medical & Scientific Officer. "We view every new grant as a potential game-changer and a signal of our passionate resolve to helping kids and adults fighting neuromuscular diseases live longer and grow stronger."

For over 65 years, MDA has funded basic, translational and clinical research. In this round of its investigator-initiated application process, MDA received 217 grant requests and awarded grants to 41 researchers, with the expectation that breakthroughs in one disease will unlock new insights and discoveries for others.  The $11 million commitment is MDA's largest such grant award in three years.

The latest round of research grants was approved recently by MDA's Board of Directors after careful deliberations and analysis by MDA's Research Advisory Committee, comprising of leading clinicians and scientists who in volunteer roles oversee the peer-reviewed process. This year MDA is funding 200 different research projects in 12 countries and 13 different diseases.

"For MDA families, medical and scientific advances can't come soon enough," added Cwik. "We are committed to helping bring safe and effective treatments and cures to those we serve as quickly as possible, and we have funded over $1 billion in neuromuscular disease research since 1950. We're able to support the best of the best, thanks to generous sponsors and supporters who champion our work."

An example of MDA's commitment to research has been evident in recent weeks, as the first possible treatments for the most prevalent form of muscular dystrophy, Duchenne muscular dystrophy (DMD), have come under review before the U.S. Food and Drug Administration. MDA has championed research in the DMD space for over 65 years and more than $209 million in funding, including supporting the foundational research that these experimental treatments and many of today's advances are built upon.

Among the new MDA research grants:

• Developing a therapy for ALS (amyotrophic lateral sclerosis): Scientists at the University of Chile will use a gene therapy approach to target a stress response pathway known as the "Unfolded Protein Response," which may yield a new therapeutic target in this devastating disease. 
• Validating a new drug target for Duchenne muscular dystrophy (DMD): Researchers at Houston Methodist Research Institute in Texas will investigate whether a new drug improves the repair ability of skeletal muscles and test its potential as a new therapy for DMD.
• Investigating potential new therapies in nemaline myopathy: Researchers at Monash University in Melbourne, Australia, will screen more than 1,200 FDA-approved drugs in a zebrafish model of nemaline myopathy to determine which are most effective at increasing muscle function and reducing disease severity.
• Testing a potential method for preventing transmission of mitochondrial myopathies: MDA funding of scientists at the Salk Institute for Biological Studies in La Jolla, Calif., will allow them to utilize an approach involving the use of enzymes to act as "molecular scissors" that are able to enter mitochondria and specifically identify and eliminate mutated mitochondrial DNA. If successful, this work could lead to the first-in-history correction of genetic diseases at the oocyte (immature egg cell) level and the potential eradication of mitochondrial diseases.
• Increasing the diagnosis rate for limb-girdle muscular dystrophy (LGMD): Scientists at Massachusetts General Hospital in Boston will work to identify new neuromuscular disease genes, which will provide deeper understanding of common disease mechanisms. Future treatment development can then be prioritized to target common mechanisms in order to benefit a larger number of patients. This project is co-funded by the American Association of Neuromuscular & Electrodiagnostic Medicine as AANEM and MDA continue to build on a partnership aimed at providing neuromuscular disease education and supporting research.

A Bold Five-Year Research Plan

In 2015, MDA announced a five-year research plan to advance frontline discoveries, including:

• Increasing MDA research spending and a refocus of its research grants program to yield even better outcomes;
• Proactively approaching the marketplace with requests for proposals for specific funding opportunities, while continuing its investigator-initiated, peer-reviewed process;
• Co-funding more grants with other interested organizations, including other voluntary health groups, government agencies and drug developers;
• Revolutionizing family care and support through its MDA Care Centers and clinical trial infrastructure. MDA has a national network of more than 150 Care Centers linked to major medical institutions, where it provides care to 235 patients daily, on average.

2016 Grants Materials

• New Research Grants Put Families at the Heart of Our Mission by Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Program Director
• Winter 2016 section in the Grants at a Glance
• Grants by the Numbers
• Grants by State

About the Muscular Dystrophy Association
MDA is leading the fight to free individuals and the families who love them from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.

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SOURCE Muscular Dystrophy Association