“Building the house” for FSHD therapies

Entrance of Colditz Castle

Patients who volunteer for research are the keystone of drug development efforts. Without them, the entire edifice built by teams of biologists, chemists, doctors, and other PhDs and MDs, would collapse. Patient involvement is more important than ever now, as many companies are starting to develop drugs. They urgently want to know, what does it mean for a drug for FSHD to be “effective”?

You might think this is a silly question. Isn’t the answer obvious? “An effective drug would repair all of my damaged muscles,” you might think. But that is a tall order and may require a treatment to regenerate new muscles using technologies like cell transplantation and tissue engineering. Such treatments are still a long ways off.

But over the next year or two, there will be drugs going into clinical trials that shut down the DUX4 gene. If they work as intended, these drugs would keep you from getting worse. Most patients say they would welcome such a treatment. Here’s the challenge: How would a drug company convince the FDA that its drug is preventing you from getting worse? If your strength test scores did not change at all over the 12 months of the trial, is that thanks to the drug, or would you have been stable even without the drug?

This is a gnarly problem, and the research needed to solve it can only be done with the participation of hundreds of patients. Right now, the ReSOLVE study, led by Drs. Rabi Tawil (University of Rochester) and Jeffrey Statland (University of Kansas), is developing two new methods to measure changes in muscle function within the timeframe of a clinical trial. The study is being run in Baltimore MD, Columbus, OH, Kansas City, Los Angeles, Rochester, NY, Salt Lake City, Seattle, WA. If you live in these areas, please consider enrolling. The more quickly people enroll, the sooner the study can be completed, and the sooner clinical trials can begin.

Several other clinical trial preparedness studies are also recruiting volunteers. To receive email alerts about these opportunities, please be sure to sign up with the FSH Society. We also list a variety of studies on our page about enrolling in a clinical study. In the video below, Dr. Jeff Statland explains why so many patients and clinical study sites are needed. Watch:

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2 responses to ““Building the house” for FSHD therapies”

  1. If you live near one of the seven CTRN sites listed in the above article, please consider volunteering for the RESOLVE study. As someone that is participating in the study, I can attest that the study does not take up much of your time… four to five visits that last about 5 hours. There are no painful procedures in the study. If you have FSHD and are reasonably mobile, you likely qualify. The study is primarily PT based, but if you qualify for the study and can’t perform certain exercises…that’s OK… you do as much as you can as well as you can. There are no drugs administered in this study.

    This is a really important study to examine various testing criteria and procedures to help assemble near future clinical trials with meaningful results. Not only that, but the study is recruiting approximately 160 FSHD patients. That’s a pretty huge number for a rare disease and is on par for what will likely be required numbers for phase 3 clinical trials. Please help to make it clear to biotechs that FSHD patients show up when needed and encourage even more biotechs to consider FSHD in their drug pipeline.

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