The CMT Research Foundation is partnering with University of Illinois Chicago’s Dr. Charles Abrams to test a potential therapeutic for CMT1X, the second most common form of CMT, behind only CMT1A in prevalence. This project will test whether treatment with inosine may ameliorate CMT1X symptoms or even change the course of the disease. Inosine is an anti-inflammatory small molecule, and its levels are reduced in animal models of CMT1X. It is hypothesized that lower inosine levels may play a role in the nerve inflammation seen in people with CMT1X and that raising inosine levels may have therapeutic benefit.

To date, Dr. Abrams and his team have identified an optimal therapeutic dose and dosing schedule for inosine in mice with CMT1X. In the next phase of the project, they will be measuring the effects of inosine treatment on the level of inflammation within the peripheral nervous system. If successful, the project will culminate in a trial of inosine therapy in mice with a genetic mutation that causes CMT1X.