In this project, researchers at AcuraStem developed a new drug-screening platform utilizing cells donated by CMT2A patients. The team used this platform to screen several thousand compounds, many of them already approved by the FDA or international regulatory agencies for use in other diseases, for their ability to promote survival of CMT2A cells. In an initial screen, thirty-seven compounds were identified as being potentially therapeutic. The AcuraStem scientists then performed a more in-depth validation stage of the project, further narrowing down the compound list to eleven that may show therapeutic benefit for people with CMT2A. While more research is needed to follow up on these leads, this project has resulted in the development of important knowledge in the continuing effort to develop treatments and cures for CMT2A.