NEW YORK (GenomeWeb News) – The National Institutes of Health will fund biomarker studies for facioscapulohumeral muscular dystrophy through a $9 million grant to the Boston Biomedical Research Institute, BBRI said Tuesday.
The funding will launch the Senator Wellstone Muscular Dystrophy Cooperative Research Center, named for the deceased Senator Paul Wellstone of Minnesota.
The Wellstone Center will work with BBRI, the FSH Society, and industry partners Acceleron Pharmaa and Genzyme to identify molecular markers for use in monitoring the effectiveness of therapeutics during clinical trials, and to start a repository of FSHD-diseased and normal muscle stem cells for use in developing drug and cell-based therapeutics.
"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," Children’s Hospital’s Genomics Program Director and Harvard Professor Louis Kunkel, who is collaborating with the center, said in a statement.
One goal of the research is to develop biomarkers that can be used to help determine the safety and effectiveness of a new class of drugs that could enhance muscle strength and mass for those suffering FSHD. The aim is that these drugs can be used to help maintain muscle strength and physical function in FSHD patients.
Other collaborators include researchers at the Johns Hopkins Hospital, the University of Sao Paolo in Brazil, the University of Maryland School of Medicine, and the University of Texas Southwestern Medical Center.