(PRWEB) July 16, 2014
NIH awards $6.3 million grant to Seattle muscular dystrophy researchers
A group of Seattle-based muscular dystrophy researchers has been awarded a prestigious $6.3 million grant from the National Institute of Health (NIH) to establish a Senator Paul D. Wellstone Cooperative Research Center.
The group will focus on translating laboratory findings into clinical treatments for two of the most common inherited muscle diseases: Duchenne Muscular Dystrophy (DMD) and FSH Muscular Dystrophy (FSHD). This is welcome news to the local community of individuals with FSHD.
Five years ago, if one asked a room full of scientists how the genetic mutation underlying FSHD caused the disease, the room would light up with fervor, imagination -- and contention. There were lots of ideas to explain why an anomalous deletion of “junk DNA” on chromosome 4 led to the progressive loss of skeletal muscle in patients with the disease, but no consensus on the cause.
In 2004, the dearth of information about the disease prompted Terry and Rick Colella to form a non-profit organization, with a mission to stimulate research on FSHD, initially funding seed projects at the University of Washington, Seattle Children’s and the Fred Hutchinson Cancer Research Center. “When our son was diagnosed 10 years ago, there had been no FSHD studies in the Seattle area,” commented the Colellas. Friends of FSH Research (Friends) has since raised over $2.5 million dollars that has funded research toward a cure for FSHD. The organization has recently been awarded Laureate status at the University of Washington for their financial contributions.
That investment has been paying off in a big way. In 2009, studies funded by Friends contributed sufficient findings to warrant a large $5 million investment by the NIH towards a Program Project Grant. This grant was awarded to a team of researchers, including Dr. Stephen Tapscott and Dr. Galina Filippova at the FHCRC, Dr. Daniel Miller at the UW, Dr. Rabi Tawil at the University of Rochester, and Dr. Silvere van der Maarel at the University of Leiden. In turn, many of those investigators, including Dr. Tapscott and Dr. Miller, now hold individual federal grants that focus on FSHD. The Muscular Dystrophy Association has also awarded large grants to Dr. Tapscott, Dr. Joel Chamberlain, and Dr. Gregory Block as a result of seed funding by Friends. Together, the 2.5 million dollar investment from Friends over 10 years has resulted in an over 6-fold return on investment.
The newly created Wellstone Center will be headed by Dr. Jeffrey Chamberlain at the University of Washington and Dr. Stephen Tapscott. Dr. Chamberlain is one of the foremost experts on Duchenne muscular dystrophy, and was the first to demonstrate that gene therapy is a viable approach to treating the disease. The grant was issued through the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a branch of the NIH and will provide the $6.3 million over a four-year period.
Friends of FSH Research is a Seattle-based non-profit organization founded in 2004. The mission of the organization is to accelerate innovative research towards a deeper understanding and treatment or cure for the disease. The organization has contributed over $2.5 million towards seed financing for projects that have earned over $15 million in federal and foundation grants. http://www.fshfriends.org