The Charcot-Marie-Tooth Association (CMTA) continues its mission to accelerate research that impacts the lives of those living with Charcot-Marie-Tooth disease (CMT). Building on previous successes, CMTA announced earlier this year a strategic $450,000 investment in a new research project to develop treatments for CMT1A and CMT1B, two of the most common forms of this debilitating disease, and could extend to other types of demyelinating CMT (CMT1), broadening the impact of this investment.
Led by Jordan VerPlank, PhD, at the Uniformed Services University of the Health Sciences, the project focuses on a new approach that could bring new hope to patients. The research repurposes an FDA-approved drug that boosts cyclic guanosine monophosphate (cGMP) levels. cGMP is an important regulator of many cellular processes. Previous CMTA-funded work by Dr. VerPlank and colleagues showed encouraging potential for CMT symptom improvement with raising cGMP levels.
In the latest phase of this research, Dr. VerPlank’s team has achieved significant progress by validating a new method to study the accumulating proteins in peripheral nerve myelin cells in CMT1A and 1B that lead to CMT symptom progression.
The team also initiated the testing of an FDA-approved drug on a model of CMT1A to determine its effectiveness in reversing or slowing disease progression. These studies are critical in bringing research closer to clinical trials.
Sue Bruhn, PhD, CMTA’s CEO, emphasized the importance of this project: “This impactful research represents a significant step towards our goal to bring effective treatments to people with CMT. By repurposing an FDA-approved drug, we can potentially shorten the timeline for bringing new therapies to our community. The encouraging developments from Dr. VerPlank’s lab highlight the potential for new treatments and reinforce the strategic vision that drives CMTA-STAR’s research initiatives.”
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