STAR, the CMTA’s research initiative, was created with one goal: to find treatments to slow, stop or reverse the progression of CMT. Working with investigators and scientists chosen from an international body of the world’s most accomplished medical pioneers, STAR is leading CMT research. The STAR program’s success is due to the willingness of scientists working in their areas of expertise to come together as a team to advance CMT research, sharing and communicating ideas, discoveries and findings. Scientists sharing their successes and failures every month is what sets the STAR program apart from any other CMT-related research effort.
The chart below outlines the steps necessary to deliver drug treatments for CMT and STAR’s progress in each type since 2008.
STAR’s collaborative research is done by university labs, large pharmaceutical companies and small research organizations working with the world’s leading CMT experts to create groundbreaking, efficient results at each step of discovery. The opportunities associated with STAR stem from the fact that the causes of CMT have been pinpointed, leading to the identification of more than 80 specific gene mutations. More importantly, the fact that these genetic mutations can be replicated in laboratory models opens an extraordinary window of opportunity to develop treatments and cures for CMT in the immediate and foreseeable future.
Building the Bridge to Faster Human Trials
Because our goal is to stop, slow or reverse the progression of CMT in people, targeted drugs must be tested in human cells. Because we cannot conduct early-stage testing in human beings, the CMTA is working with the New York Stem Cell Foundation (NYSCF) to develop human cellular models of CMT. We started with skin and blood cells from patients with different types of CMT, and banked them at the University of Iowa. These cells are being shared with the NYSCF, which transforms them into cells that can be studied in the lab. The CMTA is also collaborating with Dr. Gabsang Lee (Johns Hopkins Medical Center) and Dr. Robert Baloh (Cedars-Sinai Medical Center), who are performing research to determine the optimal cell lines for early-stage testing.
The STAR Initiative’s work is broken down by different types (CMT1A, CMT1B, CMT1X, CMT2A, CMT2E and CMT4C) and project teams consisting of experts at universities working together with scientific partners. Click on a star to learn more about that type.
The CMTA, along with the Muscular Dystrophy Association, CMT United Kingdom, and TREAT NMD (the European Neuromuscular Network), are the partnering patient advocacy groups within INC. The CMTA is well positioned to facilitate patient recruitment and involvement in cutting-edge research.
International CMT Scientific Conferences
The CMTA was a founding sponsor and collaborator of the 1st International CMT Conference in 2003. The goal was to exchange new information and, equally important, to start new collaborations and strengthen existing networks between European and North American research groups. With lectures covering clinical, diagnostic, and basic research issues of CMT neuropathies, the International CMT Consortium meetings have been held regularly since then. The 6th International CMT Consortium took place in Quebec City, Quebec, Canada in 2015.