Exciting news from a CMTA-STAR-funded research project has been published by Jordan VerPlank, PhD, Assistant Professor in the Department of Anatomy, Physiology, and Genetics at the Uniformed Services University of the Health Sciences (USU). The paper, published in Cellular and Molecular Life Sciences, reports encouraging advancements in the search for treatments for CMT1A and CMT1B.
Building on his previous CMTA-funded work in the lab of the late Laura Feltri, MD, a CMTA-STAR Advisory Board member and pioneering CMT researcher, at the State University of New York at Buffalo, Dr. VerPlank and his team discovered that raising the levels of a molecule called cGMP helps clear toxic proteins from the peripheral nerves in models with CMT1A and CMT1B. The study demonstrated that raising cGMP using CYR119, a new experimental drug developed by Cyclerion Therapeutics, improved peripheral myelin thickness, nerve function, and overall motor coordination in CMT models. This finding offers hope that similar approaches could lead to therapeutic options for patients with CMT1A and CMT1B.
In a statement, Dr. VerPlank celebrated the critical role of CMTA’s support in advancing this research: “The CMTA-STAR program has been instrumental in driving this work forward. Their continued investment has allowed us to push boundaries and explore novel treatment strategies for CMT. This publication is not just a milestone for our team but a testament to CMTA’s unwavering commitment to improving the lives of everyone affected by CMT.”
CMTA’s Chief Research Officer, Katherine Forsey, PhD, added, “This study is a shining example of the potential that CMTA-STAR’s collaborative research model can unlock. By leveraging drug mechanisms originally developed for other diseases, we’re breaking new ground in CMT research. It’s a powerful reflection of the dedication of our researchers and the strength of the CMT community whose donations have funded this work; we are all united in the pursuit of treatments and, ultimately, a cure.”
This groundbreaking research highlights the immense progress in the fight against CMT and underscores CMTA’s commitment to fostering innovative, patient-focused research. By continuously investing in collaborative efforts like these, CMTA brings us closer to a future where effective treatments—and ultimately a cure—are within reach for everyone living with CMT.
Published On: October 29, 2024