advancing muscular dystrophy research

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University of Washington
Department of Neurology

Recent News

August 2014

The Paul D. Wellstone Muscular Dystrophy Cooperative Research Center
Invites Applications for
Pilot and Feasibility Funding Conducted by Mentored Researchers

The Paul D. Wellstone Muscular Dystrophy Cooperative Research Center seeks applications from pre- and post-doctoral trainees, or other individuals in a mentored training position, for pilot and feasibility studies in basic, translational, clinical, or ethics research related to human muscular dystrophies. Projects should have direct relevance to muscular dystrophy and the mentor should have a commitment to training researchers and/or clinicians in skills needed for future muscular dystrophy research. Projects should pilot new concepts and/or test the feasibility of new approaches in muscular dystrophy research such that a successful project would provide the basis for career development and/or future progress in the area of muscular dystrophy research and care.

Deadline for application: September 15, 2014

Earliest start date and duration of the award: October 1, 2014 through April 30, 2015.

Maximum amount of each award (salary and/or supplies): $25,000.00 (including any indirect costs).

Application: Interested individuals should submit a two page application that includes these sections: 1. A description of the proposed research plan (including abstract, background/preliminary studies, aims and design). 2. A description of the mentored training. 3. A statement acknowledging the requirement for participating in ethics training and in the educational program of the MDCRC, which consists of approximately monthly meetings to discuss muscular dystrophy research. In addition to the two page application, applicants should include a Curriculum Vitae and a letter of reference from the mentor.

Please email applications, letters, and questions to: Marsha Challoner, mchallon@fhcrc.org



July 2014



May 2014

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.

 


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