By: Keith N. Fargo, Ph.D., Chief Scientific Officer, CMT Research Foundation
The CMT Research Foundation is currently funding a research project led by AcuraStem aimed at producing effective treatments for CMT2A. Using stem cells derived from adult patients, AcuraStem scientists have tested thousands of compounds for their ability to promote the survival of CMT2A nerve cells. This approach is called “drug screening” and can produce drug candidates for further testing and development in a short amount of time.
AcuraStem has completed the second phase of their three-phase project funded by the CMT Research Foundation. During phase 2, researchers identified 37 compounds that improved cell survival. Phase 3 will now study these compounds in greater detail to help scientists evaluate the best ones to take forward. Promisingly, many of the compounds they identified in phase 2 have already been approved by the FDA or international regulatory agencies for medicinal use in humans. This is exciting because so-called “repurposed” drugs may have a faster route to FDA approval and availability to people with CMT.
We will continue to keep you updated on the latest progress from this study. In the meantime, learn more about our research priorities and see all the CMT research we’re currently funding. You can also sign up to be the first to receive email updates about new research and scientific progress.
Help Advance CMT Drug Development
While no treatments or cures currently exist for CMT, the science to change that does.
Your donation to the CMT Research Foundation will fund cutting-edge research that’s solely focused on drug development and continue moving promising projects like the one at AcuraStem forward without delay. Together, we can change the course of CMT during our lifetime — and for every future generation.