The CMT Research Foundation In November 2021 awarded a grant to test a new therapeutic delivery system that may be capable of crossing the blood-nerve barrier and releasing its payload in the affected tissue without toxic side effects. Drug delivery is a fundamental problem in neurological diseases and frequently results in otherwise promising therapies failing to advance. Typically, the therapy cannot reach the nervous system or must be given in such high doses that it results in toxicity. In the peripheral nerves, this problem is caused in large part by the blood-nerve barrier, a structure that prevents many molecules from moving out of the blood stream and into nerve tissue. Overcoming this problem is significant. One way may be to package the treatment in such a way that it ‘tricks’ the peripheral nerves into allowing it entry across the blood-nerve barrier.

Kelly Langert, PhD who will be in charge of the project, is an assistant professor, Molecular Pharmacology and Neuroscience, Loyola University Chicago Stritch School of Medicine and the Research and Development Service of the Edward Hines Jr. Veterans Affairs Hospital. She will be joined in the project by her Stritch colleague, Associate Professor Virginie Mansuy-Aubert, PhD. Dr. Charles Abrams, neurologist and professor at the University of Illinois Chicago (UIC), one of the world’s foremost experts on CMT1X, and member of CMTRF’s Scientific Advisory Board, will serve as a collaborator and advisor.