New Research Grants Put Families at the Heart of Our Mission

Grace Pavlath, Ph.D. MDA Senior Vice President & Scientific Program Director
Grace Pavlath, Ph.D.
MDA Senior Vice President & Scientific Program Director

Just a little over a week ago, MDA launched a revitalized brand — one that reflects how we’re doubling down and refocusing our efforts on families. For more than six decades, we’ve been fighting to free individuals — and the families who love them — from the harmful effects of muscle-debilitating diseases. We’re fighting to give back the freedom to walk, to run. To eat. To breathe. We’re fighting to give back the freedoms that neuromuscular disease takes away.

One of the ways we’re doing this is by funding research to find effective treatments and cures. I’m happy to announce that earlier this month MDA began funding 41 new research, development and research infrastructure grants. With a nearly $11 million commitment over the next three years, these 41 new grants are now supporting investigators seeking to advance the state of science and develop effective treatments and cures in 13 of the diseases in MDA’s program.

Numbers represent progress and potential

We received 217 applications for the Winter 2016 round of grants and approved funding for close to 20 percent of them. Of note:

  • Nine of the new awards are development grants, given to young investigators who demonstrate a commitment to continuing as leaders in the neuromuscular disease research field. These grants are intended to be used as seed money to help launch the scientific programs of these promising new researchers.
  • Thirty of the awards are research grants, given to independent investigators and covering research related to therapy development for the diseases covered by MDA.
  • Two of the new awards are research infrastructure grants, which fund the development of tools, techniques or services to facilitate therapy development.
  • Awards were made to investigators in 15 states and five countries outside the U.S.

The new grants, approved in December by MDA’s Board of Directors, were recommended for funding by MDA’s Research Advisory Committee, comprising around 40 of the world’s leading clinicians and scientists who specialize in neuromuscular disease and who serve in volunteer roles for MDA. Funding of the new grants became effective Feb. 1 and will continue for two to three years, depending on the individual grant request.

With these new grants, MDA’s current research commitment totals about 200 research projects in 12 countries around the world. Each of these is a step forward toward treatments and cures.

Grants cover a broad range of diseases, impact areas

The new grants cover research into the motor neuron diseases ALS and spinal muscular atrophy. They support projects focused on the dystrophies: congenital, Duchenne and Becker, facioscapulohumeral and limb-girdle. They fund investigations into mitochondrial myopathy, nemaline myopathy, central core disease, periodic paralysis and related diseases in MDA’s program.

With these grants, we’re making progress in numerous areas, to include: increasing understanding of underlying disease mechanisms and causes, gene discovery, new research models and tools, identification and validation of new drug targets, biomarker development, therapy development, support of young investigators and collaboration with biotech and industry.

Here are some highlights:

  • Paul August, Ph.D., at Sanofi, was awarded an MDA research grant totaling $298,500 over a period of three years to develop a neuron-muscle contraction unit on a chip, using cells derived from people with amyotrophic lateral sclerosis (ALS).
  • Louis Kunkel, Ph.D., at Children’s Hospital in Boston, was awarded an MDA research grant totaling $300,000 over a period of three years to identify genetic modifiers in facioscapulohumeral muscular dystrophy (FSHD).
  • Chris Lorson, Ph.D., at the University of Missouri in Columbia, was awarded an MDA research grant totaling $300,000 over a period of three years to optimize an approach currently in clinical trials for spinal muscular atrophy (SMA) by delivering an enhanced form of drug combined with other potential synergistic therapies.
  • Ke Ma, M.D., Ph.D., at Houston Methodist Research Institute in Texas, was awarded an MDA research grant totaling $300,000 over a period of three years to investigate whether a new drug improves the repair ability of skeletal muscle and test its potential as a new therapy for Duchenne muscular dystrophy (DMD).
  • Tamar Sztal, Ph.D., at the School of Biological Sciences, Monash University, in Melbourne, Australia, was awarded an MDA development grant totaling $180,000 over a period of three years to 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.

For more information on all the new grants, check out MDA’s Grants at a Glance.

Research paves the way forward

MDA was started by families, for families. Our research program plays a critical role in helping individuals and families fighting muscular dystrophy, ALS and related life-threatening diseases live longer and grow stronger, and with this new round of grants, we’re investing in the scientists and the projects that will move us forward and help turn the hope for treatments into reality.