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Dan Proft used his Chicago’s Morning Answer program to spotlight Charcot-Marie-Tooth disease (CMT), a progressive genetic nerve disorder that affects roughly one in 2,500 people. The segment, tied to an upcoming charity golf outing at the Merit Club in Libertyville, featured Cure CMT founder Patrick Livney, who has lived with the condition since his teens.

CMT is named for the three doctors who first described it in the late 1800s. It impacts the peripheral nerves that control movement and sensation in the limbs, causing muscle weakness, atrophy, and mobility challenges. There are two main types: one that damages the protective myelin sheath around nerves and another that attacks the nerve fibers directly. In both cases, nerve signals slow or fail, leading to progressive disability. While some patients are diagnosed in childhood, others may not see symptoms until midlife or later.

Livney recalled being diagnosed at age 16, when doctors predicted he would be in a wheelchair by 30. Advances in genetic research since then have identified more than 100 mutations linked to CMT, yet there is still no approved drug therapy. Current management is limited to braces or surgery to aid mobility. “Unlike MS, which has drug cocktails that can maintain quality of life, we have nothing like that for CMT,” Livney said.

Cure CMT focuses on two goals: improving patient access to mobility aids and investing in research that could lead to the first effective treatment. Livney has been pursuing that breakthrough for 17 years and says the field has seen a surge of activity, with more than 30 pharmaceutical and biotech companies engaged. Five potential therapies are already in human clinical trials, with many more in pre-clinical stages. The most likely initial target is CMT-1A, the most common form caused by overproduction of a gene affecting myelin.

The August 18 golf outing, co-chaired by Proft, will raise funds to support Cure CMT’s mission. Livney stressed that any successful therapy—no matter which CMT type it treats first—could pave the way for broader treatments. “Any CMT that gets treated will lift the boats of all the other types,” he said.

Cure CMT Founder Highlights Progress Toward First Treatment for Debilitating Nerve Disorder