Latest research grants bring CMTA-STAR total to $17 million
GLENOLDEN, PENNSYLVANIA, USA, March 23, 2021 — The Charcot-Marie-Tooth Association | Strategy to Accelerate Research (CMTA-STAR) announced $1.1 million in grants March 23 aimed at helping top CMT scientists prepare for clinical trials for CMT1X and CMT2A.
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. Because CMT typically advances slowly, measuring progression during a multi-year clinical trial is challenging. Biomarkers measure changes over shorter periods of time than self-reported measures like clinical outcome assessments so that researchers can quickly and precisely measure whether a treatment or drug had a positive impact on neuropathy. A shortened timeline keeps costs down for companies interested in investing in CMT research.
All of the CMTA’s research efforts are consolidated under the banner of 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.
The latest grants will fund two studies that will collect patients’ natural history data over a two-year period. Dr. Michael Shy at the University of Iowa and Dr. John Svaren at the University of Wisconsin will use a $601,407 grant for a project aimed at developing outcome measures, plasma biomarkers and a nanostring platform for skin biopsy in 60 CMT1X patients. CMT1X currently affects about 15 percent of all diagnosed cases of CMT.
Another grant of $559,555 will enable Drs. Shy and Svaren to begin a project taking the same state-of-art approach to prepare for clinical trials in CMT2A, which affects about 5 percent of all patients with CMT and up to 25 percent of patients with CMT2.
The findings from the two projects will be critical in advancing clinical trials for CMT1X and CMT2A.
For more information on how to get involved in these studies, please visit cmtausa.org.
In addition to driving research forward, the CMTA provides education, resources and support to more than 70,000 patients via print publications, social media platforms, and virtual meetings.
The CMTA’s research and community services have has earned it Charity Navigator’s top 4-star rating two years in a row, as well as a perfect 100 percent score for governance and transparency for the last seven years.