The FSH Society is co-funding the second FSHD Trial Preparedness Workshop. The Workshop, held from May 29-30, 2015, at the University of Rochester Medical Center in Rochester, New York, will convene more than 50 stakeholders from around the world, including representatives from industry, the Food and Drug Administration, National Institutes of Health, and patient advocacy groups, including the FSH Society.
“For this year’s Workshop, the first objective is to reassess where we are in the process of developing relevant clinical outcome measures, biomarkers and surrogate outcome measures for future FSHD trials,” said workshop organizer Rabi Tawil, MD, of the University of Rochester. “The second goal is to reach agreement on the most promising outcome measures to be pursued and identify gaps that remain. Finally, we hope to foster collaborations among investigators at different institutions to help accelerate the pace of research to fill those gaps.”
Over the past three years, a scientific consensus has emerged around the central genetic mechanism of FSHD. While many details about the disease process remain to be solved, several labs in both academia and industry have begun to search for potential therapies. In addition, financial incentives to encourage drug development for orphan diseases, combined with advances in tools to track disease progression, have wakened industry interest in FSHD.
Anticipating rapid progress in FSHD drug development, two years ago Tawil together with Stephen Tapscott of the Fred Hutchinson Cancer Research Center in Seattle, Washington, and Silvere van der Maarel of the University of Leiden in the Netherlands, convened the first FSHD trial preparedness workshop, held in Leiden. That meeting established working groups to develop recommendations for clinical outcome measures, imaging biomarkers, and molecular biomarkers (molecules that can be detected in tissue and blood). In addition, the groups developed common protocols to prospectively evaluate and validate the various outcome measures and biomarkers across multiple sites.
The Society and two co-funders, FSHD Stichting of the Netherlands and FSHD Global Research Foundation from Australia, each donated $25,000 to cover the costs of the meeting.
“The FSH Society is proud to help fund and advise this important workshop,” said Daniel Paul Perez, FSH Society CEO and President. “Meetings like this will help all future developers of FSHD treatments design good clinical trials—and help avoid costly failures. We are investing in rigorous science, collaboration and open discussion involving all stakeholders. That’s the way to move us forward towards treatments and a cure.”
Adapted from PRWeb release.
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