by June Kinoshita, FSHD Society
The FSHD Canada Foundation, a close partner and fellow traveler of the FSHD Society since its founders Neil Camarta and Craig Kelley first met in 2010 at the Society’s annual conference, announced it has awarded a $2.8 million grant to the Clinical Trial Research Network (CTRN).
The grant includes support for the CTRN to continue the MOVE and MOVE+ natural history studies, which are aimed at generating the clinical outcome measures essential for FSHD clinical trials. “The activities funded include blood-based biomarker discovery and validation, and the addition of 50 more subjects with FSHD to the MOVE/MOVE+ natural history studies,” the foundation explained.
“The CTRN has also committed to set up clinical trial sites in Canada,” the foundation said—thrilling news for the FSHD Society’s Ontario chapter, which held its first Walk & Roll to Cure FSHD in 2021 to raise awareness and funds for the Canadian initiative. The major portion of the grant comes from Solve FSHD, a recently established Canadian company focused on accelerating drug development for FSHD. Solve FSHD’s Executive Director, Eva Chin, PhD, will be presenting an FSHD University webinar this March to introduce the company to our community.
The FSHD Canada Foundation grant is a fantastic example of how the FSHD Society’s pioneering investment in the CTRN has been multiplied many times over, with game-changing impacts. The largest-ever natural history studies of FSHD are currently running on the network. Many pharmaceutical companies have told us that the existence of the CTRN was a key factor in their decision to develop FSHD therapies.
It all began at a 2013 workshop in Leiden, the Netherlands, where key research leaders including Stephen Tapscott, Rabi Tawil, and Silvere van der Maarel invited a few dozen colleagues (as well as the FSHD Society) to brainstorm about preparing for clinical trials and establish working groups. Two years later, an expanded group including officials from the FDA, met again, this time in Rochester, NY, and produced a blueprint. The FSHD Society rallied FSHD Stichting of the Netherlands and FSHD Global Research Foundation to join it in co-funding this pivotal meeting.
Soon after, Rabi Tawil, MD, of the University of Rochester along with Jeffrey Statland, MD, of the University of Kansas, obtained funding from a private donor to launch a four-site FSHD Clinical Trial Network which was then significantly expanded with support from the Society. “The principal objective is to create sites ready to do clinical trials in FSHD and to validate new outcome measures,” Tawil explained. “One the obstacles is the absence of any funding to do this. We realize that this is not something that most funding agencies will take on easily. The establishment of the FSHD CTRN with the support of the Society was critical in obtaining NIH funding for the ReSolve study.”
This was the precisely the type of gap that the Society seeks to overcome. Indeed, the Society specifically asked the CTRN to establish a key administrative position that was critical for getting the network started. The Geraldi Norton Foundation and an anonymous donor in Texas quickly responded to our request for funding to cover this grant. Another donor provided additional funds, and the CTRN was formally launched in 2016 with seven sites in the U.S.
Since its launch, the CTRN has received major funding from the National Institutes of Health and the Muscular Dystrophy Association, as well as from Friends of FSH Research and other donors. Dyne Therapeutics funded the expansion of the network to European sites. In addition, companies like Acceleron, Fulcrum, and Avidity have funded important research studies and clinical trials involving CTRN sites. With the core network well-supported, the FSHD Society was able to provide additional grants for further expansion. Today, the CTRN comprises 12 U.S. and 6 European locations.
“The CTRN is a critical component for clinical trial readiness,” said Jamshid Arjomand, PhD, chief science officer of the FSHD Society. “We are delighted to share the news of FSHD Canada Foundation’s funding to support vitally important studies and expand the CTRN to sites in Canada. Our Canadian families have been hoping to participate in research and trials for many years. Because many stakeholders worked together, their dream is now a reality.”