The Charcot-Marie-Tooth Association’s Strategy To Accelerate Research (CMTA-STAR) is CMTA’s global initiative advancing treatments for Charcot-Marie-Tooth disease (CMT). Since launching in 2008, CMTA-STAR has united patients, clinicians, researchers, and industry partners to drive research forward and bring real solutions closer to reality.
What Does CMTA-STAR Do?
CMTA-STAR accelerates progress by funding high-impact research, developing preclinical tools, and building collaborations across science and industry. Every project is guided by one goal: to bring treatments to people with CMT as quickly, safely, and effectively as possible.
CMTA-STAR’s Core Principles
- Focus on the Biology: All strategies begin with understanding the genetic cause of CMT and identifying where interventions are most likely to succeed.
- Fund Top Scientists: We identify leading CMT researchers and fund targeted projects aligned with our treatment-focused goals.
- Ensure Accountability: Projects are milestone-driven. We fund outcomes, not ideas, and hold researchers accountable to deliver.
- Foster Collaboration: CMTA-STAR breaks down silos by connecting investigators across institutions and disciplines to share tools, insights, and discoveries.
- Build Strategic Partnerships: Through CMTA-STAR, we work with industry partners to move therapies through development. CMTA-STAR provides the infrastructure; they carry it through to market.
CMTA-STAR Advisory Board Leadership
The CMTA-STAR Advisory Board brings together world-leading experts across three core areas:
- Scientific Advisory Board (SAB): Provides scientific input and guidance for research projects
- Clinical Expert Board (CEB): Supports clinical trial readiness, planning, and delivery
- Therapy Expert Board (TEB): Evaluates translational potential of research and therapeutics
This expert guidance helps move discoveries from the lab into real-world testing.
Our Research Focus
CMTA-STAR supports more than 50 active research projects across every type of CMT. Many are designed with cross-type potential, meaning they could benefit multiple subtypes. Key focus areas include:
- Genetic therapies: ASOs, gene replacement, and gene editing (CRISPR)
- Delivery systems: AAV9 vectors, nanoparticles, and other targeted methods
- Biomarkers: Tools to measure treatment impact across subtypes
- Drug development: Including the rapid progression of CMT-SORD to Phase III clinical trial
The CMTA-STAR Preclinical Toolbox
To support faster, more efficient therapy testing, CMTA has developed preclinical toolboxes designed to accelerate research across a wide range of CMT types. These toolboxes provide ready-to-use resources for researchers and industry partners, including:
- In vivo and in vitro drug profiling assays
- Patient-derived stem cell lines
- Validated animal models
- Licensing and access under partner-friendly terms
Toolboxes are available for CMT1A, CMT1B, CMTX1, CMT2A, CMT2E, CMT2Y, CMT4A, and CMT4J. Visit the CMTA-STAR Preclinical Toolbox to learn more.
Five Key Elements Driving CMTA-STAR
In addition to its guiding principles, CMTA-STAR is built on five key research elements that form the foundation of our collaborative scientific model:
- Assays: Laboratory tests that model CMT biology and allow rapid screening of potential treatments.
- Animal Models: Disease models that replicate CMT symptoms and enable real-world testing of new therapies.
- Stem Cell Models: Patient-derived Schwann cells and neurons that bring human biology into the research pipeline.
- Industry Collaboration: A robust partnership network that brings experimental therapies into CMT-specific testing platforms.
- Clinical Trial Readiness: Infrastructure that supports recruitment, biomarker validation, and trial delivery through the CMTA-Inherited Neuropathy Consortium (INC) Alliance and the CMTA Centers of Excellence network.
A Growing Network of Partners
CMTA-STAR’s collaborative model brings together more than 30 pharmaceutical, biotech, and service companies. These partners work with CMTA-funded investigators and specialized labs to evaluate treatments efficiently and move promising therapies into clinical development.
Together, We Drive Progress
The generosity and participation of the CMT community power CMTA-STAR’s impact. Every breakthrough, from gene discovery to clinical trials, is made possible by community donors and our patient partners who believe in a future without CMT.
Your support accelerates research, drives treatment development, and brings hope to everyone with CMT.
