The Muscular Dystrophy Association has awarded 44 grants totaling $13.5 million to support research efforts aimed at advancing understanding of disease processes and uncovering new strategies for treatments and cures of muscular dystrophy and the more than 40 other diseases in the Association’s program.
The new grants were reviewed by MDA’s Scientific and Medical Advisory Committees, and approved by MDA’s Board of Directors at its December meeting.
“MDA-supported researchers have long led the way in advancing understanding of, and developing therapeutic strategies for, neuromuscular diseases,” said noted pediatrician, human biochemical geneticist and MDA Chairman of the Board R. Rodney Howell.
“With this most recent round of grants and the researchers and projects they fund, the Association stands to add significantly to the ever-growing collection of knowledge that brings us, day by day and step by step, closer to the therapies and cures that will put an end to devastating neuromuscular diseases.”
About the new grants
Of the 44 new awards, 37 are primary research grants meant to improve understanding of the causes of neuromuscular diseases or guide the development of strategies for their diagnosis and treatment.
The newly awarded research grants will support research into general muscle disease, ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease), Becker muscular dystrophy (BMD), centronuclear/myotubular myopathies (CNM) and (MTM), Charcot-Marie-Tooth disease (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), Friedreich’s ataxia (FA), general muscle disease, inclusion-body myositis (IBM), Lambert-Eaton myasthenic syndrome (LEMS), limb-girdle muscular dystrophy (LGMD), mitochondrial myopathies (MITO), myasthenia gravis (MG), myotonia congenita (MC), myotonic muscular dystrophy (MMD, or DM), spinal muscular atrophy (SMA), and spinal-bulbar muscular atrophy (SBMA).
In addition to the research grants, two of the new awards are designated clinical research training grants (CRTGs) and are designed to provide promising young physicians with the research training opportunities needed to become effective clinical investigators in neuromuscular disease research.
The two CRTG grants were awarded to James Berry at Massachusetts General Hospital in Boston, and Araya Puwanant at the University of Rochester in New York. The grants will support each awardee’s completion of a two-year fellowship, during which Berry will study the experimental drug ISIS-333611 in human clinical testing of individuals with familial, or inherited, ALS, and Puwanant will study the disease process in MMD.
Five career development grants also were included in the overall count. These grants are designed to increase the number of outstanding scientists working on neuromuscular disease research. Awardees work in the laboratory of a senior investigator; each is given the flexibility to work independently or as part of a collaborative effort.
Those who were awarded development grants include:
“MDA is committed to finding cures and therapies for the diseases in its program,” said Valerie Cwik, MDA executive vice president for research and medical director. “It’s through research projects funded by grants such as those newly awarded by the Association that will help ensure we accomplish that goal.”
Information on a few of the 44 grants awarded by MDA is listed below. To see information on all the new grants, visit MDA's Grants at a Glance slideshow.
Duchenne muscular dystrophy (DMD): MDA awarded $527,670 over three years to Dongsheng Duan at the University of Missouri in Columbia to help support Duan’s research into gene therapy in DMD. Duan has worked extensively in the past with adeno-associated virus (AAV) “vectors,” the empty shells of viruses used to encapsulate healthy genes (or modified — in this case, shortened “mini-” or “micro-genes”) which are then injected locally or systemically in research model test subjects undergoing gene therapy treatment. In his latest work, Duan and colleagues will test both a new AAV delivery vehicle and a new DMD micro-gene, first in a mouse model of DMD and later in another animal research model of the disease. The investigators will evaluate whether the novel combination ameliorates DMD symptoms in test subjects.
MDA awarded $220,000 over two years to Jen-Chywan Wang at the University of California, Berkeley to research the effects of chronic glucocorticoid treatment, commonly used in DMD to delay or relieve symptoms. Although the drugs, which are thought to block inflammation, are beneficial, long-term use can cause unwanted side effects, including muscle deterioration. Wang’s study team will first investigate the means by which glucocorticoids cause muscles to degenerate. The group also seeks to determine whether it’s possible to use chemical compounds to block the muscle deterioration caused by glucocorticoids without affecting their anti-inflammatory effects.
Myotonic muscular dystrophy (MMD or DM): Mani Mahadevan at the University of Virginia in Charlottesville was awarded $435,000 over three years to research the interactions inMMD1 (also called DM1) between particular proteins and RNA (the chemical step between DNA instructions and protein production), using muscle cells from both research mouse models and MMD1-affected individuals.
Friedreich's ataxia (FA): Joseph Sarsero at Murdoch Childrens Research Institute in Parkville, Victoria, Australia, was awarded $416,250 over three years to develop a new research mouse model for FA. The model will contain an entire human gene and harbor the same disease-causing mutation as found in humans with FA. This new research tool is expected to enable scientists to better understand the molecular underpinnings of the disease as well as provide a better test subject for potential FA therapeutics.
Muscular dystrophies: Xiaohua Wu at Carolinas Medical Center in Charlotte, N.C., was awarded $224,863 over three years for research into effective therapies for various types of muscular dystrophy (MD). Wu and colleagues plan to screen more than 100,000 small compounds that might help enhance muscle function. The group will then test the most promising compounds in laboratory cell culture studies and in research mouse models of MD for potential advancement to therapeutic development.
General muscle disease research: MDA awarded a grant totaling $369,165 over three years to Bradley Olwin at the University of Colorado in Boulder. The funds will help support Olwin’s study of muscle regenerationin injured and diseased skeletal muscle. Olwin is a longtime MDA grantee, having received funding from the Association almost continuously since the early 1980s. In previous studies, Olwin and colleagues identified a rare stem cell that they hypothesize is a primary source of skeletal muscle stem cells. Olwin now plans to study the newly identified cells and identify any contribution they may make in regenerating tissue in injured and diseased skeletal muscle.