BOSTON, Sept. 10 /PRNewswire-USNewswire/ -- The U.S. National Institutes of Health (NIH) have awarded $9 million to launch a unique collaboration of researchers, clinicians, patients, government research agencies and pharmaceutical/biomedical companies to study the causes and potential treatments for facioscapulohumeral muscular dystrophy (FSHD), a muscle weakening and disabling disease that affects, at the least, one in 20,000 individuals worldwide. The award will create the first Senator Wellstone Muscular Dystrophy Cooperative Research Center (MD CRC) to focus on FSHD, the award recipients announced today.
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Headquartered at the Boston Biomedical Research Institute (BBRI), this center for excellence in muscular dystrophy will be the first ever in New England, and the first of these prestigious centers -- named for the late Senator Paul Wellstone -- to focus on FSHD, the second most prevalent adult muscular dystrophy. The Wellstone MD CRCs themselves represent a paradigm shift in research because of their intensely collaborative nature and particularly because of their mandate to include the patient advocacy organizations as a full partner in the research process.
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"We see this as a unique opportunity to tackle a tough disease in the most efficient way to set the stage for development of therapies," says Dr. Charles Emerson, director of the new center and president of BBRI. "We have recruited the best minds in basic and clinical research and have an extraordinarily committed patient advocacy group to help us focus on this problem."
Co-director, Dr. Louis Kunkel, a professor of Genetics and Pediatrics at Harvard Medical School, director of the Genomics Program at Children's Hospital and an investigator with the Howard Hughes Medical Institute, agrees. "We believe this research center model will bring discoveries from bench to bedside more rapidly than the traditional model, because we already have the full participation of the people with the most at stake in our work -- the patients," he says.
In fact, both Emerson and Kunkel admit that without the advocacy of Daniel Paul Perez, FSHD patient, resident of Bedford, MA, and the founder and CEO of the FSH Society, the new Wellstone Center might not have become a reality. According to the two scientists, Perez has been fighting tirelessly behind the scenes and in front of Congress, the NIH and the scientific community for 20 years for funds, increased research and attention for this disease. "This is a victory for all of us," says Perez. "It represents decades of work by dedicated researchers, Society leaders and patients to find a research home for this disease." Perez was diagnosed as a toddler and has been the national leader for patients with FSHD since graduating from college.
Another major collaborator will be Acceleron Pharma, a biotech company that will partner with the Wellstone Center scientists and clinicians to determine the safety and effectiveness of a new class of drugs that enhance muscle mass and strength. The hope is that these drugs will help maintain muscle strength and physical function in patients with FSHD and other dystrophies.
Additionally, Genzyme Corporation will participate in the development of cell-based therapeutic approaches, which also have noteworthy promise.
Economically, the Wellstone Center will have a positive impact, according to Bob Coughlin, president of Massachusetts Biotechnology Council, who believes it is just the kind of program that will strengthen the regional economy and the life science industry in Massachusetts. "The new center at Boston Biomedical Research Institute will be a magnet for drawing talent into the state," says Coughlin,
