Using Fingolimod to Treat CMT

The Martini laboratory has also initiated a program of evaluating drugs approved for other diseases for their possible use in treating CMT. The principle being followed is that drugs with demonstrated positive effects in related diseases can be rapidly re-positioned to treatment of CMT disorders following preclinical studies in rodent models of CMT. This approach aims at leveraging scientific knowledge of CMT disease pathology to identify potentially useful drugs that are already available, and perform enabling animal studies that can accelerate their application in the treatment of CMT patients.

The drug Fingolimod (Gilenya) is FDA approved for the treatment of Multiple Sclerosis, a demyelinating disorder affecting the brain. In a TIN-sponsored study newly published in Neuromuscular Disorders (https://doi.org/10.1016/j.nmd.2025.105345) the Martini group now reports that CMT1X mice treated with Fingolimod showed improved nerve function, and the drug positively changed some motor functions (balance), as well as grip strength. The results suggest that Fingolimod, or more advanced drugs of this class now also available, may be a treatment option to ease the disease burden in severe cases of CMT1X. The approach may also be of use in other Type 1 CMT’s in which immune system dysfunction is similarly involved.