GLENOLDEN, PA — The Charcot-Marie-Tooth Association (CMTA) has announced a pioneering international research initiative, committing over $500,000 to two multiyear projects aimed at developing treatments for the most prevalent demyelinating forms of Charcot-Marie-Tooth disease (CMT): CMT1A and CMT1B. The significant investment underscores CMTA’s dedication to improving the lives of those affected by CMT and finding a cure.
Leading the first project is Professor John Svaren, Ph.D., Chairperson of the CMTA Scientific Advisory Board and based at the University of Wisconsin. His team will explore innovative pathways for decreasing PMP22 expression in CMT1A using a small molecule approach. Overexpression of PMP22, a protein encoded by the PMP22 gene, is the root cause of CMT1A. Svaren’s laboratory aims to develop an orally administered drug that could normalize PMP22 levels in Schwann cells, potentially improving disease symptoms and severity.
“If we’re successful, the study will identify a new agent for lowering PMP22 levels that would allow for rapid testing in human clinical trials in CMT1A,” said Professor Svaren.
The second project involves a gene-editing approach, using the CRISPR-Cas9 system. This revolutionary technology could potentially provide a universal treatment for both CMT1A and CMT1B patients, regardless of their specific mutations. Professor Svaren will collaborate with Maurizio D’Antonio, Ph.D., at the San Raffaele Scientific Institute in Milan, Italy, and Bruce Conklin, M.D., at the Gladstone Institute in San Francisco, CA.
“It’s an exciting opportunity to explore the potential of genome editing in targeting CMT1A and CMT1B, anticipating optimal strategies for these common CMT types,” Professor Svaren said.
“Demyelinating CMTs affect over half of all people with this devastating disease,” explained Katheriene Forsey, Ph.D., the CMTA’s Chief Research Officer. “This latest investment through the CMTA’s Strategy to Accelerate Research (STAR) reflects our multi-modality approach to tackling CMT head-on. Through partnerships with world-leading research scientists and projects with rapid translational potential, we continue to accelerate progress towards the development of treatments.”
The groundbreaking effort by the CMTA is a beacon of hope for those affected by CMT, offering the possibility of innovative new treatments and, ultimately, a cure.
For the latest news on everything happening in Chester County and the surrounding area, be sure to follow MyChesCo on Google News and MSN.