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MDA Awards $2.6 Million in New Research Grants

Investment funds critical infrastructure, feeds a rapidly expanding therapy development pipeline and encourages researchers and other stakeholders to commit to the neuromuscular field

CHICAGO, April 26, 2018 – The Muscular Dystrophy Association today announced the award of 13 new research, development, and research infrastructure grants totaling $2.6 million and targeted to impact the state of neuromuscular science and accelerate the development of treatments for muscular dystrophy and related life-threatening diseases. The investment reflects MDA’s commitment to fund the best, most promising science in an effort to find life-changing and lifesaving treatments and for individuals living with neuromuscular disease.

“MDA is pleased to announce our support of more than a dozen new research projects as part of our efforts to keep up the momentum during this time of unprecedented progress in neuromuscular disease research and care,” said MDA President & CEO Lynn O’Connor Vos. “Each and every project we fund has the potential to result in improved understanding of the causes and underlying mechanisms of the neuromuscular diseases under MDA’s umbrella, as well as findings that can inform the development of therapies that will transform lives.”

Of the new grants, five were awarded to established, independent investigators; five were awarded to investigators at the beginning of their careers and on the brink of becoming independent investigators; and three are supporting the development of tools, techniques or services to facilitate therapy development.

For more than six decades, MDA has funded basic, translational and clinical research as it works to unlock the mechanisms of neuromuscular disease and enable scientists and clinicians to develop safe and effective therapies capable of changing lives. The power of MDA’s research program lies in its big-picture approach that leverages insights and information resulting from research in one disease area to inform and advance discoveries and breakthroughs in others. The 13 new awards were made to leading scientists who are conducting innovative research targeted to make an impact across all the diseases in MDA’s program.

The latest round of research grants was approved by MDA’s Board of Directors following careful deliberations and analysis by MDA’s Research Advisory Committee, through which leading clinicians and scientists in volunteer roles oversee the peer-review process.

Vital research and services dollars come from generous MDA partners and supporters who organize, and donate to, community fundraising programs including the International Association of Fire Fighters (IAFF), CITGO Petroleum Corporation, Harley-Davidson Motor Company, Acosta, Albertsons Companies, Casey’s General Stores, Circle K, The Kroger Company, National Association of Letter Carriers and Dutch Bros. Coffee.

“MDA continues to fund the top researchers and the most promising science in the neuromuscular disease space,” said MDA Senior Vice President & Scientific Director Grace Pavlath, Ph.D. “With these new awards, we’re advancing projects that are shedding light on the causes of disease; putting into place infrastructure and key elements for clinical trial readiness and completion; informing and driving the development of cutting-edge therapies; and fostering collaboration and partnerships between academia, pharma and other key stakeholders in the search for solutions that will change lives.”

Currently, MDA is funding about 160 different research projects around the world with a combined investment of nearly $50 million, raised by MDA’s supporters.

Among the new MDA awards:

Lukasz Sznajder, Ph.D., a postdoctoral associate at the University of Florida College of Medicine in Gainesville, Fla., was awarded an MDA development grant to apply next-generation sequencing and other technologies to develop novel and disease-specific blood biomarkers for “repeat expansion diseases” (diseases that are caused by the abnormal expansion of particular stretches of DNA); These include ALS and frontotemporal dementia (FTD) caused by a mutation in C9ORF72 and type 2 myotonic dystrophy (DM2).

Kristi Wharton, Ph.D., professor of biology at the Brown University Institute for Brain Science in Providence, R.I., was awarded an MDA research grant. In this new academic-industry partnership, Wharton is working to identify novel drug targets for ALS (amyotrophic lateral sclerosis) that might quickly be developed using the resources available at Pfizer.

MDA and the Target ALS Foundation announced a partnership in September 2016. Now, MDA has partnered again with Target ALS to provide $100,000 in support to advance the collaborative work of the ALS Biomarker Collaboration.

Stephan Zuchner, M.D., Ph.D., chairman of the Dr. John T. Macdonald Foundation of Human Genetics at the University of Miami School of Medicine in Florida, was awarded an MDA research infrastructure grant to expand and make more widely available resources to streamline gene identification for diagnostics and therapy development efforts for Charcot-Marie-Tooth disease (CMT).

Marta Margeta, M.D., Ph.D., associate professor of pathology at University of California San Francisco School of Medicine, was awarded an MDA research grant to improve understanding of the cellular and molecular pathways by which dysregulation of a process called autophagy impairs skeletal muscle function and leads to muscle injury in sporadic inclusion-body myositis (IBM) and other neuromuscular diseases.

Daniel MacArthur, Ph.D., co-director of medical and population genetics at the Broad Institute of Harvard and MIT in Cambridge, Mass., was awarded an MDA research infrastructure grant to create a neuromuscular disease-specific platform for the Rare Genome Project, which will provide more individuals living with neuromuscular diseases access to a definitive diagnosis, through advanced genome sequencing techniques.

Robert Bryson-Richardson, Ph.D., associate professor at the School of Biological Sciences, Monash University, in Victoria, Australia, was awarded an MDA research grant to investigate potential approaches to treating myofibrillar myopathy (MFM) caused by a mutation in the Filamin C gene.

Winter 2018 Grants Materials

• 13 New MDA Grants Totaling $2.6 Million Push for More Progress, by Grace Pavlath, Ph.D., MDA Senior Vice President & Scientific Director
• MDA Grants at a Glance

MDA has funded more than $1 billion in neuromuscular disease research since 1950. In just the last three years, four drugs have been approved by the U.S. Food and Drug Administration (FDA) that can trace their origins directly to MDA funding. These successes illustrate the vibrancy of MDA’s research program and the welcoming of a new era in treating neuromuscular diseases.

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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