New center to design therapies for muscular dystrophy

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New center to design therapies for muscular dystrophy

Goal is to bring promising lab treatments into patient trials
Leila Gray

A newly established muscular dystrophy research center in Seattle hopes to bring promising laboratory results into therapeutic trials. The center will design and test treatments for the most common forms of the muscle-wasting disorder, Duchenne muscular dystrophy and facioscapulohumeral dystrophy, also called FSHD. 

Jeffrey Chamberlain
In mice with muscular dystrophy, green indicates successful delivery of gene therapy to muscle cells in the sample. The detail image shows muscle stem cells (light blue), supporting cells (dark blue) and blood vessels and another type of stem cell (red).
images of restored muscle cells in mice treated for muscular dystrophy

Muscular dystrophy interferes with the growth and repair of skeletal muscle, and causes progressive loss of physical ability. Movement and mobility declines, and lives can be cut short if breathing muscles fail. Because there is no cure, treatment options for people with the disease have been limited.

Now, after years of scientific studies, several potential methods for managing the disease are on the horizon.

“Our plan is to develop a clinical infrastructure to advance cures for muscular dystrophy,” said Dr. Jeffrey S. Chamberlain, the McCaw Chair in Muscular Dystrophy and professor of neurology at the University of Washington. He co-directs the center with Dr. Stephen Tapscott, an investigator at the Fred Hutchinson Cancer Research Center and a UW professor of neurology. Scientists and clinicians from their institutions will collaborate with those from Seattle Children’s and University of Rochester (NY).

Previous studies led by Chamberlain have shown that gene therapy can eliminate Duchenne muscular dystrophy in rodent models of the disorder. He and his colleagues are working to apply those findings to clinical applications for patients with the disease. Other studies will seek better rodent models for FSHD to adapt the gene-therapy strategies for a wider range of muscular dystrophies. 

The new center also will support patient studies on the causes and mechanisms behind the disease's progression.

Jeffrey Chamberlain, UW professor of neurology, co-directs the new center.
picture of Jeffrey Chamberlain

 One of the first of these clinical trials will be an imaging study to explore inflammation and markers of disease progression in FSHD.  Tapscott led earlier studies that elucidated the cause of FSHD. The present work aims to advance that knowledge into clinical application. As an initial step, a magnetic resonance imaging, or MRI, study of individuals with the disease will be led by Dr. Dennis Shaw and Dr. Leo Wang at the University of Washington and Seattle Children’s, with Dr. Rabi Tawil at the University of Rochester, to evaluate and validate biomarkers in the disease.

Duchenne muscular dystrophy and FSHD are both among the most common inherited human disorders. Duchenne muscular dystrophy primarily affects children; FSHD more commonly first appears during adolescence or adulthood. While a defective gene critical for muscle strength causes Duchenne muscular dystrophy, a toxic protein produced in muscle causes FSHD. 

The new effort is one of the nation’s Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Centers. It is funded through four-year, $6.3 million grant  from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, a branch of the National Institutes of Health.

The National Institutes of Health, Seattle’s Friends of FSH Research, the Muscular Dystrophy Association, Edgar Martinez, and the Pistol Creek Research Foundation supported the preliminary studies leading to the formation of the new center. 

News editors: Interviews are available with Jeffrey Chamberlain. Media contact for this story is Elizabeth Hunter, 206.616-3192,