There is an urgent need for new treatments and therapeutic approaches for Wolfram syndrome, including both oral and injectable medications. A promising strategy involves targeting upstream disease mechanisms to delay the progression of symptoms associated with Wolfram syndrome, such as optic nerve atrophy, neurodegeneration, and diabetes, through the use of oral medication.
In pursuit of this goal, we have collaborated with Amylyx Pharmaceuticals in Cambridge, Massachusetts, to develop a novel oral medication, AMX0035. AMX0035 aims to address endoplasmic reticulum stress and mitochondrial dysfunction, which are upstream molecular mechanisms underlying Wolfram syndrome. We recently published an article about this project in a leading medical journal, which can be accessed for free at the following link: https://insight.jci.org/articles/view/156549.
In late 2020, the US FDA granted orphan drug designation to AMX0035 for treating Wolfram syndrome. Using longitudinal study data and dantrolene clinical trial data, we’ve developed a clinical trial plan and carefully crafted our protocol to ensure patient safety and accurate assessment of AMX0035’s effectiveness. We’re excited to share that our trial protocol has received a positive response from both the US FDA and the Washington University Medical Center Institutional Review Board. This development brings us one step closer to starting our clinical trial for Wolfram syndrome treatment.
If you’re interested in participating in this trial, please reach out to our research nurse coordinator, Stacy Hurst, at 314-747-3294.
With hope,
Fumi Urano, MD, PhD
Samuel E. Schechter Professor of Medicine
Washington University Medical Center