We are pleased to announce that NIH has awarded a Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center for research in Seattle and Rochester. This is a collaborative venture between scientist and clinicians at the University of Washington, Fred Hutchinson Cancer Research Center, Seattle Children’s Hospital and The University of Rochester. The Center will be supported by a $6.3 million award from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a branch of the National Institutes of Health (NIH).
Dr. Jeffrey Chamberlain at the University of Washington is the Director and Dr. Stephen Tapscott at the Fred Hutchinson Cancer Research Center is Co-director of the Wellstone Center, which will conduct research projects in the areas of Duchenne muscular dystrophy (DMD) and Facioscapulhumeral dystrophy (FSHD), the two most common forms of inherited muscle disorders.
Research on DMD will focus on advancing methods for using Gene Therapy to treat the muscular dystrophies. Previous studies led by Dr. Jeffrey Chamberlain have shown that gene therapy can be used to eliminate muscular dystrophy in rodent models of the disorder, and the present work is designed to advance the studies for clinical application. The focus is on using “micro-dystrophin” genes as a way to treat DMD. Dr. Chamberlain’s lab pioneered the development of micro-dystrophins, and recent studies suggest they represent an extremely promising method for long-term treatment of all cases of DMD and the related Becker muscular dystrophy. The DMD gene therapy studies will refine methods for whole body gene delivery to eliminate disease symptoms in limbs, breathing muscles and the heart. These studies will involve Drs. Chamberlain, Stephen Hauschka, Guy Odom and Martin Childers (U.W. Neurology and Rehabilitative Medicine) as well as Dr. Zejing Wang (FHCRC).
Further studies will focus on improving rodent models for FSHD and adapting the gene therapy strategies for a wider range of muscular dystrophies, particularly FSHD and other dominant disorders. These latter studies are led by Dr. Joel Chamberlain (U.W. Department of Medicine).
A major focus of the Center will be on developing a clinical infrastructure at the University of Washington, Seattle Children’s Hospital and the University of Rochester to support human clinical trials of treatments, including gene therapy, for various types of muscular dystrophy.
One of the first clinical trials to be supported will be an imaging study to explore progression, inflammation and markers of disease progression in FSHD. Pioneering studies led by Dr. Tapscott have helped to define the cause of FSHD, and the present work aims to advance that knowledge into clinical application. The present work will advance those discoveries by conducting a Magnetic Resonance Imaging (MRI) study of individuals with the disease, led by Dr. Dennis Shaw and Dr. Leo Wang at the University of Washington and Seattle Children’s Hospital with the combined participation of Dr. Rabi Tawil at the University of Rochester, to evaluate and validate biomarkers present in the disease.
DMD and FSHD are progressive, debilitating muscle disorders with no cure and very limited treatment options. Both are among the most common inherited human disorders, with DMD primarily affecting children and FSHD primarily affecting teens and adults. While DMD is caused by a defective gene critical for muscle strength, FSHD is caused by production of a toxic protein in muscle.
By developing translational and clinical trial approaches to both disorders the members of the Center hope to bring novel treatments for many types of muscular dystrophy into clinical application at the University of Washington.
Preliminary studies leading to the Wellstone Center award have been supported by the NIH, the Muscular Dystrophy Association and several private foundations. These latter organizations include the Bruce McCaw Endowment, the Edgar Martinez Golf Classic, Friends of FSH Research and the Harmonize for Hope Foundation. Additional details on the Wellstone Center will be forthcoming.