Charcot-Marie-Tooth disease progressively damages the peripheral nerves, causing muscle weakness, loss of sensation, and difficulty walking. There’s no approved treatment. That’s exactly why the CMT Research Foundation exists, and what we’re working to change.
We’ve recently been in the news with Alan Jackson’s Last Call Tour donating a portion of each ticket sold to support CMT research through our organization.
We’re grateful for every dollar (you can help here!) that goes directly to support scientific research like this:
- Tracking CMT with a smartphone (Columbia University). One of the biggest barriers to running effective clinical trials is that CMT changes slowly and subtly, and current measurement tools aren’t sensitive enough to detect it without years of observation. Dr. Wolfgang Pernice’s DANCER platform uses AI to extract detailed 3D movement data from a simple smartphone video, potentially letting patients and researchers track disease progression from anywhere in the world.
- Smarter drug delivery (Loyola University Chicago). One of the trickiest problems in CMT research isn’t just finding potential therapies; it’s getting them to the nerve where they can make a difference. Dr. Kelly Langert’s team engineered tiny nanoparticles coated in immune cell membranes, essentially disguising them so the body delivers them straight to affected nerve tissue. In pre-clinical models, these targeted particles accumulated in diseased nerves while leaving healthy tissue alone. This project has reached its final research milestone, and the next step is loading those nanoparticles with therapeutic cargo and testing them in additional CMT models.
- Finding the best genetic treatment for CMT2A (UC San Diego). CMT2A is caused by mutations in a single gene, but over 100 different mutations in this gene cause CMT2A, making a one-size-fits-all approach hard. Dr. Uri Manor’s team is reprogramming patient skin cells into nerve cells, using AI-enhanced microscopes to identify a consistent fingerprint of cellular damage, then running head-to-head comparisons of genetic medicines to find the strongest candidate for clinical trials.
- A blood test to tell if treatments are working (Johns Hopkins). Clinical trials for CMT2C are on the horizon, but researchers need a simple, reliable way to know if a drug is working effectively . Drs. Jeremy Sullivan and Charlotte Sumner discovered that the faulty protein behind CMT2C causes certain markers to leak into the bloodstream. Their team is developing a blood-based test that could rise and fall in step with disease activity, a critical tool with the potential to make trials faster and more conclusive.
Every one of these projects is funded by donors. The science is real, the momentum is real, and the finish line is closer than it’s ever been.
