Supported by angel donors and industry partners, our Therapeutic Accelerator Project promises to overcome obstacles to FDA approval and ensure that effective therapies get to our families by 2025.
Since the genetic disease mechanism was discovered in 2010, facioscapulohumeral muscular dystrophy (FSHD) research has picked up speed and caught momentum.
Brilliant researchers are developing therapies that improve—and sometimes reverse—the condition in a test tube, but there is a lack of accepted criteria to prove that a drug is effective in patients and deserving of FDA (and European EMA) approval. This is beginning to slow the drug development process down. Overcoming the hurdles is the only way to shorten the time it will take to get these therapies to our families. With every affected patient in mind, we have risen to this challenge and started our Therapeutic Accelerator Initiative.
In March 2019, the FSHD Society convened a landmark meeting attended by FDA regulators as well as researchers from seven biopharmaceutical companies, academic scientists who are leading the effort to prepare the field for clinical trials, and representatives of patient advocacy organizations including the MDA, Friends of FSH, and Chris Carrino Foundation. The workshop introduced the FDA to FSH muscular dystrophy–the impact of the disease on individuals, why scientists think FSHD is treatable, and the progress that has been made toward clinical trials–and sought input from the FDA on how to develop methods to measure treatment effects to meet the standards for FDA approval. The findings from the workshop are summarized in a white paper, Industry Collaborative Workshop for Therapy Development in FSHD.
Therapeutic Accelerator Project areas
All of these areas require a coordinated effort to ensure that clinical trial measurement tools meet industry and FDA standards. All drug development companies will have an accepted way to measure the effectiveness of their drugs, and the most promising will go into clinical trials faster.
Genetic diagnosis. Our genetic diagnostics project is identifying state-of-the-art technologies and expertise to develop a faster, more cost-effective genetic test.
Molecular biomarkers. We will develop a blood test that biopharmas can rely on to measure the effectiveness of their drugs in clinical trials.
Imaging biomarkers. While promising as a way to measure whether a drug is working, magnetic resonance imaging (MRI) findings must be confirmed and proven to correlate with other measures such as strength and function.
Integrating global patient registries & natural history studies. We are working on ways to aggregate and analyze data across multiple registries to gain a more rigorous, comprehensive view of the condition.
Patient-focused Drug Development meeting. In June of 2020, the FSHD Society convened the landmark externally-led patient-focused drug development (EL-PFDD) meeting on FSHD. Testimony from hundreds of patients has been released in our Voice of the Patient Report.
Anybody can do the
ordinary…We are being
challenged to do the
- Mark Stone
President and CEO