The CMTA’s new $200K+ investment accelerates CMT1A organoid research, revolutionizing treatment evaluation for Charcot-Marie-Tooth disease (CMT)

GLENOLDEN, PENNSYLVANIA, USA, February 21, 2024 — The Charcot-Marie-Tooth Association (CMTA), the largest philanthropic funder of Charcot-Marie-Tooth disease (CMT) research aimed at bringing treatments and a cure to patients, announced today an investment of more than $200,000 in new funding to develop an innovative organoid model of CMT1A, the most common form of this disease. This cutting-edge approach will enable scientists to efficiently evaluate potential treatments that target defects in myelin, the protective sheath around peripheral nerves.

Led by Professor Vincent Timmerman, Ph.D., the Peripheral Neuropathy Research Group at the University of Antwerp in Belgium is spearheading a project aimed at assessing CMT1A organoids to accelerate drug testing. Dr. Timmerman is a pioneer in the creation of these miniature organs from induced pluripotent stem cells (iPSCs). “As we recently developed human-derived miniature organ-like structures, here named organoids,” said Dr. Timmerman, “we highly appreciate the support of the CMTA which will allow us to characterize this system for evaluating CMT1A neuropathy. The availability of a three-dimensional structure will allow a qualitative and quantitative assessment of candidate therapies enhancing preclinical studies.”

“As we embark on this pioneering project to develop organoid models for CMT1A, we’re not just addressing a singular aspect of the disease; rather, we’re contributing to a larger framework of research aimed at revolutionizing how we understand and treat CMT,” said Katherine Forsey, Ph.D., CMTA Chief Research Officer. “By investing in cutting-edge methodologies like organoid modeling, the CMTA is not only accelerating progress toward effective treatments for CMT1A but also laying the groundwork for advancements across multiple types of CMT. This strategic investment underscores our commitment to leveraging innovative approaches and collaborative partnerships to drive meaningful impact for CMT patients worldwide.”

Published: February 21, 2024