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 stage of their three-stage project funded by the CMT Research Foundation. During stage 2, researchers identified 37 compounds that improved cell survival. Stage 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 stage 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.