GLENOLDEN, PA, UNITED STATES, September 28, 2023 – The CMTA Board of Directors approved a $206,018 grant to the Inherited Neuropathy Consortium (INC) for additional work to prepare for clinical trials, including outcome measures and biomarkers, at four of INC’s 20 sites.
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.
INC is an integrated group of academic medical centers, patient support organizations and clinical research resources dedicated to conducting clinical research on different forms of CMT and improving patient care. Funded primarily by the National Institutes of Health (NIH), with supplemental funding from the CMTA and the Muscular Dystrophy Association, INC plays a key role in developing the infrastructure necessary to evaluate CMT therapies.
INC is currently working with pharmaceutical and academic partners on multiple clinical trials that are underway or in the planning stages, including CMT1A, CMT1B, CMT2A, CMT1X and other rarer forms such as SORD recessive neuropathy.
Outcome measure and biomarker studies are “fundamental to bring clinical trials to CMT,” grant reviewers said, adding that “longitudinal assessments in the ongoing studies are critical and completion of these studies in a rigorous fashion is a very high priority for translational development.”
Noting that INC is the only group capable of performing the proposed projects, CMTA reviewers said it is “highly probable” that the grant will lead to a patent, license, external funding through biomarker development, new gene discoveries and therapeutic clinical trials.
With previous CMTA support, INC enrolled almost 7,000 patients into its registry and developed CMT-specific clinical outcome assessments (COA) to measure disability in adults and children with CMT. INC also identified several biomarkers, including MRI imaging, skin biopsies and plasma markers, and applied wearable sensors to obtain sensitive gait and balance data during activities of daily living, yielding a “digital biomarker” with enhanced responsiveness to change. INC’s earlier work has reduced the typical clinical trial risks of not achieving expected outcomes or lacking the statistical power to obtain meaningful results.
Ongoing biomarker studies funded by the CMTA (for CMT1B, CMT1X, CMT2A, and CMT2F) depend on the INC infrastructure. Reviewers said that while the scope of the proposed plan is large, INC has a track record of success and will recruit personnel to spearhead many of the wide-ranging initiatives: The grant will fund at least six full-time trainees to become independent investigators in CMT-related research; expand the number of full-time trainees and provide training to additional postdoctoral fellows and students.
All the CMTA’s research efforts are consolidated under the banner of its Strategy to Accelerate Research (STAR), which brings together the world’s largest network of biotech research partners, research scientists, clinicians and patients. The CMTA funds more CMT research than any other philanthropic organization, more than $18.5 since 2008, with plans to invest another $10 million in the next few years. The CMTA also funds a wide array of patient services, including a summer camp for kids with CMT, an international network of support groups and multidisciplinary Centers of Excellence where patients receive treatment and participate in research.
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Kenny Raymond
Head of Communications, Charcot-Marie-Tooth Association
