MDA Establishes Myotonic Dystrophy Clinical Research Network

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MDA has launched a new, five-center clinical research network focused on type 1 and type 2 myotonic muscular dystrophy (MMD1 and MMD2, also known as DM1 and DM2), with the principal goal of preparing for testing of new MMD treatments as they become available.

Each center in the new network has a particular interest and expertise in MMD, and the centers are distributed across the United States to maximize opportunities for participation in studies. Neurologist Charles Thornton at the University of Rochester Medical Center is the network's overall principal investigator.

The researchers in each center will work together to standardize methods of evaluating disease severity and will determine the best ways to assess whether experimental treatments are beneficial.

The new MMD centers are:

• California: Stanford University School of Medicine, Stanford; John Day, M.D., Ph.D. (director)
• Florida: University of Florida College of Medicine, Gainesville; Tetsuo Ashizawa, M.D. (director)
• Kansas: University of Kansas Medical Center Research Institute, Kansas City; Richard Barohn, M.D. (director)
• New York: University of Rochester Medical Center, Rochester; Charles Thornton, M.D. (director)
• Ohio: Ohio State University Medical Center, Columbus; John Kissel, M.D. (director)

MMD network a response to scientific progress

"Recent years have seen rapid progress in the development of molecularly targeted therapy for myotonic dystrophy," said Jane Larkindale, MDA's director of translational research.

"Basic research has shown that this disorder involves a disease mechanism known as 'RNA toxicity,' and translational research has shown that this mechanism is unusually responsive to therapeutic intervention. Based on the current pace of therapeutic development, it is reasonable to expect that new therapies will be advanced into clinical trials in the near future, and the new network will ensure that we're prepared for that."

Similar networks for ALS, DMD, CMT

In 2009, MDA established clinical research networks for amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy (DMD) through five MDA/ALS centers and five MDA clinics with special expertise (see Speeding the Course of Clinical Trials). In 2011, the Association partnered with other groups to form a Charcot-Marie-Tooth disease (CMT) network, which continues to receive MDA support (see Providing a Network for Clinical Research in CMT).

For more information

To learn more about MMD1 and MMD2, see:

• MMD Research: Seeking to Free Proteins from a 'Toxic Web' (Quest article describing three different research strategies in MMD, published July 2012); and
• In Focus: Myotonic Muscular Dystrophy (Quest series discussing clinical aspects of MMD, published April 2012).