Researchers ID Potential Treatment for Form of Muscular Dystrophy

The University of Virginia Medical Center is making strides in finding a treatment for myotonic dystrophy, which the most common type of muscular dystrophy.

The potential treatment is an experimental drug currently used to treat other conditions, such as rheumatoid arthritis. In lab tests, mice with myotonic dystrophy that were given this drug had better muscle function and improved strength.

Doctors are hopeful that this could also be applied to humans.

"The goal here would be to block the inflammation that is occurring in the muscles because it appears that that inflammation, the longer it goes on in these chronic diseases, the more it leads to muscle degradation and muscle break down," said Erin Foff, assistant professor at the UVA Medical Center Department of Neurology.

The drug will now need to go through clinical trials with myotonic dystrophy patients.

CHARLOTTESVILLE, Va., March 17, 2015 A doctor who was one of the discoverers of the gene responsible for myotonic muscular dystrophy has now identified a therapeutic that could slow progression of muscle damage and muscle dysfunction associated with the disease issues that cause patients significant disability and deterioration in quality of life.

The potential treatment is an experimental drug currently being evaluated for treating other conditions, such as rheumatoid arthritis. In lab tests, mice with myotonic dystrophy that were given the treatment had better muscle function in tests such as running on a treadmill and had improved grip strength. In addition, their muscles became healthier and, notably, many even lived longer. While more testing needs to be done, Mani S. Mahadevan, MD, of the University of Virginia School of Medicine, is hopeful about the drug's potential in humans as well.

The nice thing about this therapy is that we know that it's already been shown to be safe, because clinical trials have already been done with it for other conditions. That's a big, big hurdle that's been overcome, Mahadevan said. With a lot of drugs, the problem is that once you do these proof-of-concept studies, the drugs need to be developed a lot further, refined and tested for safety and efficacy. But a lot of that work has been done, so therefore we can leapfrog the development of this therapy so that it can be moved into clinical trials sooner.

Understanding Myotonic Dystrophy

Mahadevan, of UVA's Department of Pathology, has been conducting pioneering research into the causes of myotonic dystrophy, the most common form of muscular dystrophy, for more than two decades. His work revealed that the condition is caused by an expanding piece of DNA a mutation that grows worse with each generation. Many people, he noted, do not even realize they have the mutation until a child or grandchild is born with a severe form of the disease.

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Researchers ID Potential Treatment for Form of Muscular Dystrophy