Our community has been promised for decades that “one day in the future” there will be treatments for FSHD, and so it’s amazing to realize that day is almost upon us. The future is NOW for clinical trials. In 2019, we saw two Phase 2 trials—for Acceleron’s ACE-083 and Fulcrum’s ReDUX4 (losmapimod). A dozen biopharma companies and academic groups are working hard on the next wave of therapies, and we anticipate more clinical trials over the next few years. It’s very important that we use what we have learned so far to speed up the process and get treatments to our families as soon as possible!
Bottom line: we need to double the number of patients who are actively engaged, especially younger and moderately affected individuals. While companies are investing millions of dollars into research and trials, they can’t succeed without us. Our community must engage fully with the process. Across all research, 85 percent of trials face delays and 30 percent never even get off the ground because of the shortage of volunteers. This dramatically slows research, which means our families must wait longer for treatments.
This summer, Fulcrum Therapeutics launched its Phase 2 ReDUX4 trial of losmapimod, the first drug which has been shown to reduce the expression of a gene called DUX4 that causes FSHD. In the lead-up to this trial, Fulcrum conducted a “biomarker study” that is designed to evaluate the reliability of different measures of efficacy that will be used in the drug trial. We helped to recruit volunteers by sending emails to patients in the vicinity of the research centers involved in the study. We contacted nearly 700 patients and family members, and some 100 people responded. In the end, the study enrolled around 20 volunteers, just enough to complete the study, but it took several months longer than anticipated.
Why was it so difficult for this critical study to recruit enough patient? Researchers need to start with a well-defined population in order get a good signal from a study or trial. If patients are too diverse in their age, disease severity, etc., this can make the data difficult to interpret. For this reason, volunteers had to pass through many hoops to qualify. They had to be the right age. They had to be willing and able to complete multiple visits to the clinic over several months. They needed to be moderately affected—not too little, but also not too much. They had to have an actively affected leg muscle (identified by MRI.). They needed to be willing to have two muscle biopsies of that same muscle (one at the beginning and one at the end of the trial), because muscle tissue is the only method currently available to accurately measure DUX4 expression and the potential effect of losmapimod on the root cause of disease.
Given these complexities, it’s easy to understand why some volunteers did not meet the requirements for the study. The ReDUX4 clinical trial is now under way and must recruit 66 volunteers, who must all pass through the same stringent requirements. Every study and drug trial will face similar challenges. And this is why we need many more of you—patients and family members—to be actively engaged and join our “standing army” that is ready to leap into action.
The time is now. The need is urgent. Here’s what you can do:
- Join our contact registry for research;
- Make sure our emails to you aren’t going into junk mail folders;
- Read our e-newsletters and alerts to keep yourself up to date.
Learn how your contribution to our year-end campaign helps to speed up clinical trials.