Record-breaking interest highlights progress, desire to find breakthroughs across diseases

CHICAGO, August 25, 2015 – Powered by its big-picture perspective to accelerate treatments and cures across the broad spectrum of neuromuscular diseases, MDA today announced the award of $10 million in new research grants to the world’s brightest scientists conducting leading-edge discovery for muscular dystrophy, ALS and related muscle-debilitating diseases.

Through its investigator-initiated application process, MDA received 350 grant requests, the most ever in its 65-year history of funding basic, translational and clinical research. It awarded grants to 36 researchers, half new to MDA, with the expectation that breakthroughs in one disease will unlock new insights and discoveries for others. Clinical research saw the greatest area of funding growth, a deliberate effort by MDA to fund more work tied to therapy and drug development.

“These new grants are potential game-changers, a signal of our passionate resolve to helping kids and adults fighting neuromuscular diseases live longer and grow stronger,” said Valerie Cwik, M.D., MDA’s Executive Vice President and Chief Medical & Scientific Officer. “There’s more new drug discovery underway than ever before, and we’re determined to double the number of promising human clinical trials in the next five years.”

MDA’s long-term investment in research has contributed to more than 30 clinical trials in the past year alone for novel drugs and other therapies aimed at treating a broad spectrum of neuromuscular diseases. The latest round of research grants were approved recently by MDA’s Board of Directors after careful deliberations and analysis by MDA’s Medical and Scientific Advisory Committees comprised of leading clinicians and scientists who in volunteer roles oversee the peer-reviewed process. This year MDA is funding 250 different research projects in 16 countries.

“We’re supporting the best of the best, thanks to generous sponsors and supporters who champion our work,” Cwik said. “For MDA families, advancements can’t come soon enough.”

Earlier this year, MDA announced a bold, five-year research plan to advance frontline discoveries, including:

• An increase in MDA research spending and a refocus of its research grant 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 clinics and clinical trial infrastructure. MDA has a national network of more than 150 clinics linked to major medical institutions, where it provides care to 235 patients daily, on average.

Watch Louis M. Kunkel, Ph.D., in the first installment of MDA’s Research Minute where he discusses the importance of funding new investigators and bringing them to the neuromuscular disease field.

Among the new MDA research grants:

• Understanding and developing therapies for ALS (amyotrophic lateral sclerosis): Scientists at the University of Kentucky in Lexington will study a new drug target for ALS and test a potential therapy for the disease. About $2.2 million of the new research grants target ALS.
• Validating a new and exciting drug target for Duchenne muscular dystrophy (DMD): Researchers at Children’s Hospital in Boston will determine how a protein called Jagged1 prevents the symptoms of DMD in animal models bearing the mutation that causes the disease.
• Exploring a new target for facioscapulohumeral muscular dystrophy (FSHD): Stanford University scientists in Palo Alto, Calif., will build on our latest understanding of the disease and test a therapeutic strategy for treating it.
• Expanding therapeutic possibilities in Friedreich’s ataxia (FA): Scientists at the University of Oklahoma Health Sciences Center will work to find the optimal HDAC inhibitor and dose for increasing production of the frataxin protein, which is deficient in FA and, in parallel, test an alternative HDAC inhibitor that may prove more effective than others tested to date.
• Studying disease mechanisms and pursuing treatments in congenital muscular dystrophy (CMD): MDA funding of scientists at Columbia University in New York will allow them to study the underlying mechanisms that drive a recently-discovered form of CMD and test three possible treatment paradigms.
• Testing a new therapeutic strategy in spinal muscular atrophy (SMA): Scientists at Johns Hopkins University School of Medicine in Baltimore will test a gene therapy strategy to increase SMN protein levels. This project is co-funded by the American Association of Neuromuscular & Electrodiagnostic Medicine as AANEM and MDA build on a partnership aimed at providing neuromuscular disease education and supporting research.

Strength in Numbers: New Grants Pave the Way Toward Treatments and Cures by MDA Senior Vice President and Scientific Program Director Grace Pavlath, Ph.D.
MDA Summer 2015 Research Grants By the Numbers Infographic
MDA Summer 2015 Grants by State
Summer 2015 Grants at a Glance
About MDA

The Muscular Dystrophy Association is the world’s leading nonprofit health agency dedicated to saving and improving the lives of people with muscle disease, including muscular dystrophy, amyotrophic lateral sclerosis (ALS) and other neuromuscular diseases. It does so by funding worldwide research to find treatments and cures; by providing comprehensive health care services and support to MDA families nationwide; and by rallying communities to fight back through advocacy, fundraising and local engagement.

Visit mda.org and follow us at facebook.com/MDAorg and @MDAorg. Learn more about MDA’s mission by watching this video.