CMTA Awards $100,000 to Test Treating CMT1X with Common Food Additive

GLENOLDEN, PENNSYLVANIA, USA, May 25, 2021 / — The Charcot-Marie-Tooth Association announced a $100,000 grant May 25 that will fund a study into whether a common food additive can be used to treat CMT1X.

John Svaren, PhD, of the University of Wisconsin (the head of the CMTA’s Scientific Advisory Board) and investigators at the Jackson Laboratory will use a mouse model of CMT1X to perform the studies, which will take about a year. If successful in demonstrating proof of concept, these studies could lead to an accelerated path to the clinic.

CMT is a degenerative neuromuscular disease that kills the long, or peripheral, nerves to the hands and feet. As the nerves die, the muscles around them follow suit. All of the CMTA’s research efforts are consolidated under its Strategy to Accelerate Research, or STAR—which brings together the world’s largest network of biotech research partners, research scientists, clinicians and patients—and funds more CMT grants than any other philanthropic organization to increase the likelihood of finding a cure. Since 2008, the CMTA has invested more than $17 million in STAR, with plans to invest another $10 million in the next few years.

CMT1X is a demyelinating CMT that accounts for roughly 15 percent of all cases. It is caused by mutations in the gap junctions that form channels that allow transport of metabolic substances across the many layers of myelin. Recent studies have highlighted the important role of metabolic exchange between the axons of nerves and the myelin sheath made by Schwann cells, and the lack of functional channels could be the key factor that drives the deterioration of axons in this type of CMT.

Investigators will test whether the food additive can prevent metabolic starvation and maintain healthy nerves when channels are blocked by the mutations that cause CMT1X. The food additive has been proven to be safe at even high doses in preclinical/clinical studies.

Marcia Semmes
Charcot-Marie-Tooth Association
+1 443-631-1859
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