CMTA backs research on when to treat CMT1A and CMT1E

The Charcot-Marie-Tooth Association is investing $165,000 in University of Iowa research aimed at improving treatment timing, biomarker development and clinical trial design for CMT1A and CMT1E. The work could help determine when therapies targeting PMP22 are most likely to preserve nerve function.

Why it matters: - The CMTA investment is aimed at answering a key question for families and drug developers: when treatment for CMT1A and CMT1E should begin to have the best chance of preserving nerve function. - The project also seeks biomarkers that could show whether a therapy is working before physical changes can be measured, which could make clinical trials faster and more informative. - CMT1A and CMT1E are among the common forms of Charcot-Marie-Tooth disease, and both are tied to PMP22 biology.

What happened: - The Charcot-Marie-Tooth Association announced a $165,000 research grant on Sept. 1, 2026. - The project is led by Michael Shy, MD, and Robert Piper, PhD, at the University of Iowa. - The research will develop preclinical mouse models for CMT1A and CMT1E. - The models will be used to study the impact of PMP22 activity at different stages of disease progression.

The details: - In CMT1A, an extra copy of PMP22 causes the body to make too much PMP22 protein. - In CMT1E, mutations damage peripheral nerve myelin early in life. - PMP22 is essential for healthy peripheral nerve myelin, the protective sheath around nerves. - The new models are designed to help determine the optimal time for therapeutic intervention. - The models will also aim to identify biomarkers that can signal response before functional improvement is visible. - CMTA said the work supports CMTA-STAR, its Strategy to Accelerate Research focused on drug development for common CMT subtypes. - Shy said therapies targeting PMP22 are advancing, but clinical trials still need answers on when to start treatment, how much correction is needed and how to measure nerve response. - Katherine Forsey, CMTA’s chief research officer, said validated models and early biomarkers can help researchers and industry partners design better trials and evaluate therapies earlier in disease.

Between the lines: - The funding reflects a shift from broad disease description toward trial-ready tools that can help move candidate therapies into the clinic. - The emphasis on early biomarkers suggests CMTA is trying to reduce the time and uncertainty between lab results and patient-facing treatment decisions. - The project builds on a four-year collaboration between Shy and Piper. - Their prior work includes multiple peer-reviewed studies in CMT and myelin biology, including a 2025 natural history study of CMT1E in Brain that found progression is fastest during childhood and adolescence and linked PMP22 biology to disease severity. - CMTA said it has invested more than $33 million in CMT research since 2008.

What's next: - The University of Iowa team will use the new funding to build and test mouse models that can support future therapeutic studies. - The research is expected to inform how future clinical trials for PMP22-targeting therapies are designed and timed. - CMTA’s broader research push will continue to focus on tools that can move promising science toward treatments and, eventually, a cure.

The bottom line: - The grant is not a treatment result yet, but it could help define the timing, measurement and biology needed to get CMT1A and CMT1E therapies into better clinical trials.