The CMT Research Foundation funded a collaborative research partnership with Dr. Maurizio D’Antonio who made several important discoveries in MPZ, including the identification of the UPR disease mechanism that can be modulated thanks to a compound named IFB-088. Currently in testing in a phase 1 clinical trial sponsored by Inflectis BioScience, IFB-088 could advance to a phase 2 clinical trial for CMT1B. 

This project tested the genetic modulation of the same pathway in adult animal models with CMT1B to determine if treatment with the compound later in life may modify the course of the disease. In middle aged mice, the therapy significantly improved nerve conductivity and motor function. In elderly mice these improvements were lost, indicating that a more aggressive treatment approach may be necessary in older people with CMT. Additional preclinical testing is needed to further optimize the therapy prior to clinical trials. In the future, the compound may be able to slow or stop disease progression in people who’ve suffered from symptoms of CMT1B for decades.