With CMTA support of $281,339, researchers at Albany Medical College, led by Sophie Belin, PhD, and Yannick Poitelon, PhD, are exploring the newly discovered TEAD1 pathway in Schwann cells that may regulate PMP22, the gene at the root of CMT1A. By modulating this pathway with molecules previously under development, the team aims to determine whether this “control switch” can reduce excess PMP22 protein that leads to CMT1A and restore normal peripheral nerve myelin function.
The three-year study will evaluate the potential of repurposing these molecules to accelerate progress toward treatments, with a focus on directly targeting the underlying biology of CMT1A. This work builds on previous CMTA-funded research conducted in the Feltri-Wrabetz lab at the State University of New York at Buffalo.
December 2025 Update
The team has now evaluated two candidate compounds, VT107 and VT103, for their ability to lower Pmp22 gene expression in Schwann cells. VT107 produced a clear dose-dependent reduction (higher dosing led to more reduction) and showed a stronger and more sustained effect on the Schwann cell–specific Pmp22 promoter. VT103 also reduced Pmp22 expression, but the effect was shorter-lived.
These findings distinguish the durability and performance of the two compounds and identify VT107 as the more robust modulator of Pmp22 expression in Schwann cells. The results are guiding refinement of dosing strategies and supporting ongoing studies to link changes in gene expression to downstream effects on PMP22 protein levels and cellular function.
