CMT Treatment: A New Drug Joins the Fight Against Charcot-Marie-Tooth Disease
In the laboratory of theCharles Gerhardt Institute in Montpellier (UM) and the Molecular Mechanisms in Neurodegenerative Dementias (UMInserm/EPHE), a team of scientists is developing a molecule designed to target a receptor involved in several rare diseases. The goal: to open up a novel therapeutic avenue against Charcot-Marie-Tooth disease. A project supported and guided by the Montpellier University Innovation Cluster.
Ultimately, the Cure CMT project could transform the lives of thousands of patients who have reached a therapeutic dead end… For several years, alongside his colleagues Benjamin Delprat, Tangui Maurice, Tahar Ayad, and Jean-Luc Pirat, David Virieux, a researcher at the Charles Gerhardt Institute of Montpellier (ICGM), has been developing molecules that target the Sigma-1 receptor, a key protein involved in numerous rare and neurodegenerative diseases. He hopes to thereby curb the most debilitating symptoms of several neurodegenerative diseases, including Charcot-Marie-Tooth disease type 2 (CMT2A), on which he is actively working.
But to better understand the significance of this approach, we need to take a moment to look at their previous research on Wolfram syndrome, which affects about 100 people in France. In 2022, in close collaboration with the Molecular Mechanisms in Neurodegenerative Dementias (MMDN) laboratory, ICGM researchers developed the very first molecule targeting this incurable disease, for which patients had previously had no therapeutic options.
“We have demonstrated the benefits of this candidate in vivo for the most prominent symptoms … At this stage of our research, will we be able to halt the progression of the disease? We need to continue this work, but we hope, at the very least, to slow it down sufficiently and significantly improve patients’ quality of life.”
David Virieux
At the same time, following the filing of an initial patent, the two teams also succeeded in demonstrating that this same molecule could have a beneficial effect on Alzheimer’s disease. “We demonstrated that it was possible to positively affect short-term and long-term memory in an advanced animal model,” explains the researcher, who co-founded Sitera Pharmaceuticals with his partners in 2025, with the goal of launching clinical trials by 2028.
Building on these early successes, David Virieux and his team then turned their attention to Charcot-Marie-Tooth disease, a genetic disorder characterized by peripheral nerve dysfunction, decreased muscle strength, and loss of sensation, which affects between 30,000 and 50,000 people in France.And barely launched, the project, dubbed Cure CMT, hit the mark. In October 2024, it was named a winner of the Montpellier Innovation Booster (BIM), a flagship initiative of the University Innovation Hub, and received a grant of 30,000 euros. On this occasion, David Virieux received support from BIM through coaching sessions to help him present and explain a complex scientific project. “We worked with students from the master’s program at Montpellier Management (MOMA), who specialize in innovative project management. They helped us clarify our message. As scientists, we tend to get bogged down in complicated concepts… There, I learned to simplify and communicate through gestures. Imagine the various neurodegenerative diseases as the five fingers of my hand; we can target a specific receptor and thus address a disease. We can also choose a receptor common to all these conditions, which could be represented by my wrist, so that a single drug could have the ability to influence multiple diseases, ” says the researcher . “Our goal is to promote the idea that it’s possible to address multiple conditions at once, and thus convince pharmaceutical companies to partner with us on these projects.”
Following the BIM grant, which was used to expand their team dedicated to the Cure CMT project, Sitera was named the national winner of the I-Lab innovation competition. This recognition and strong support will enable them to take another step toward the clinical trial phases. “For Charcot-Marie-Tooth disease, our model isn’t advanced enough yet, but the goal remains the same: to provide a solution for patients and improve their quality of life,” explains David Virieux. This year, the researchers will therefore attempt to validate their proof of concept using a more advanced animal model—the zebrafish—which is “more relevant for mimicking how a drug behaves in humans.” The road ahead is long, but if all goes smoothly, the team hopes to begin clinical trials by 2030.