A few years ago, we told our donors that a gift to the FSHD Society “is one of the most powerful investments you will ever make to advance medical research.” It might have sounded like hubris, but we meant it. And today, we see that our bold claim is truly justified.
By the middle of next year, there will be three candidate drugs in Phase 2 or 3 clinical trials. Nearly 20 companies have an FSHD drug development program. Some of the world’s largest pharmaceutical companies have entered the ring. Some are still in “stealth mode.” The infrastructure for FSHD research has also mushroomed. There are nearly two dozen FSHD Clinical Trial Research Network (CTRN) sites across the U.S., UK, Canada, and Europe. Patient advocacy groups are taking root across the world and growing in capability and influence.
How did we come so far so fast? We used our leverage. Small research grants, “seed funding” awarded to smart, capable scientists, led to projects that could leverage major funding from the National Institutes of Health, the Muscular Dystrophy Association and its counterparts in France, Italy, the UK, the Netherlands, and beyond.
Similarly, when we funded the workshop and cornerstone grant to help launch the CTRN, we created an entity that the NIH and MDA could now support through multimillion-dollar infrastructure and project grants. The CTRN became the platform for conducting natural history studies and clinical trials, which attracted additional resources from biopharmaceutical companies. Recently, the FSHD Canada Foundation invested some $2 million to further expand the CTRN. The Society’s total investment to date of $1 million has led to $10 million in funding, not even counting pharmaceutical sources, a 10-to-1 leverage.
In another example, a few years ago we supported research by a small biotech, miRecule, to provide proof-of-concept for their approach to treating FSHD. The company has leveraged its research to cement an agreement with pharma giant Sanofi, which will pour substantial resources into advancing miRecule’s technology. Our initial $150,000 award to miRecule moved things forward faster and resulted in a potential $40 million-plus investment from Sanofi.
Knowing how to apply the levers
What’s our secret to success? First, we know where we stand: with patients. Everything we do is scrutinized through the lens of will this benefit the FSHD Community? Will it speed up more treatments to our families? Second, we have an incredible team that has deep understanding of rare disease and drug development. We know where to apply the levers. Third, we collaborate with everyone. We don’t care where the funding comes from or who is funding what, as long as it serves our mission. As our CEO Mark Stone says, “we just want more and better for our families faster.”
But mostly, we succeed because of you. To everyone who has volunteered, taken part in surveys and studies, organized chapters and fundraisers, attended our meetings, and donated, your efforts are integral and essential to the Society’s success. It is by working together that we wield the levers to move the world.