The Muscular Dystrophy Association recently awarded an MDA clinical research network grant to develop and maintain a core facioscapulohumeral muscular dystrophy (FSHD) Clinical Trial Research Network (CTRN).
The investment, totaling $1.2 million over three years, supports seven medical centers that specialize in FSHD research and clinical care and is targeted to spur advances in FSHD research and speed the development of new therapies for FSHD.
An urgent need for infrastructure
Recent breakthroughs in research include the identification of the underlying molecular cause of FSHD: the abnormal activation of a gene called DUX4, which normally is silenced or inactive. Armed with this knowledge, a number of drug companies have begun working to develop and test targeted therapeutic strategies to treat FSHD. Consequently, an urgent need exists to establish the tools that will be necessary to execute high-quality clinical trials.
“MDA is pleased to support the expansion of this critical infrastructure for facioscapulohumeral muscular dystrophy research and therapy development,” MDA Scientific Program Officer Laura Hagerty, Ph.D., said. “Today is FSHD Awareness Day, and it’s exciting to see and share with the FSHD community the progress taking place, with a rapidly growing pipeline of FSHD drugs in development.
“In order to keep that pipeline moving, however, it’s critical to ensure that clinical trials can be conducted quickly and efficiently to test these potential new treatments,” Hagerty added. “MDA support will enable the FSHD Clinical Trial Research Network to build a team of trained clinical evaluators all working with standardized approaches and develop regulatory strategies optimized to streamline drug trials.”
Closing gaps
Successful FSHD clinical trials depend on several factors, including the ability to recruit patients, patient access, a precise understanding of the natural history of FSHD and the major contributors to its variability, and reliable outcome measures that are sensitive to change in FSHD.
“Meetings with industry leaders, advocacy groups and FSHD researchers have identified several gaps in our clinical trial arsenal and have pinpointed clinical trial planning as a major goal for the community,” said principal investigator Jeffrey Statland, M.D. “Not only will the FSHD CTRN help close gaps in trial readiness, but a network of sites will be created with a centralized streamlined regulatory process, specific common expertise in FSHD and an engaged patient population ready to conduct efficient, high-quality clinical trials.”
The network comprises seven medical centers with significant expertise in FSHD research and clinical care. Centers are:
- California: University of California Los Angeles — Perry Shieh, M.D., Ph.D., site principal investigator
- Kansas: University of Kansas Medical Center — Jeffrey Statland, M.D., site principal investigator
- Maryland: Kennedy Krieger Institute — Kathryn Wagner, M.D., site principal investigator
- New York: University of Rochester Medical Center — Rabi Tawil, M.D., site principal investigator
- Ohio: The Ohio State University — John T. Kissel, M.D., site principal investigator
- Utah: University of Utah — Nicholas Johnson, M.D., site principal investigator
- Washington: University of Washington — Leo H. Wang, M.D., Ph.D., site principal investigator
MDA is committed to finding treatments and cures for FSHD
MDA has funded more than $22 million in FSHD research since 1950, and including this most recent award, currently is funding 10 active FSHD grants (with another two pending), with a total funding commitment of more than $3 million.
The grant to support the FSHD CTRN was approved by MDA’s Board of Directors after careful deliberations and analysis by MDA advisors and research staff. Currently, MDA is funding 177 research projects around the world, with another 28 pending.
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