CMTA is excited to report significant progress in its strategic CMTA-STAR investment of over $200,000 towards developing a human-derived organoid model for CMT1A. This pioneering research led by Professor Vincent Timmerman, PhD, at the University of Antwerp in Antwerp, Belgium, aims to accelerate drug testing by creating organoids—three-dimensional structures mimicking peripheral nerve cells—that will allow researchers to evaluate treatments targeting myelin defects in CMT1A.
In the first six months of the project, the team focused on optimizing the development of these peripheral nerve organoids, which contain the key cell type found in human peripheral nerves: Schwann cells. A major breakthrough has been in visualizing Schwann cell interactions with axons in real-time, using advanced fluorescence microscopy techniques. These Schwann cells are responsible for myelination, the process by which they form a protective sheath around axons, a crucial function impaired in CMT1A.
This new imaging technique allows researchers to observe myelination processes without labor-intensive and costly transmission electron microscopy (TEM), speeding up the analysis of potential therapies. In parallel, the team has been refining methods to produce organoids more consistently and reproducibly, ensuring they can reliably model the effects of various treatments.
Looking Ahead
With these promising early results, the project’s next phase will further optimize the organoid models for therapy testing. Dr. Timmerman and colleagues are also working to expand these methods for other forms of CMT, laying the groundwork for a broader application of this innovative approach. The ultimate goal is to create a standardized, scalable system for evaluating candidate therapies, which will help accelerate research for CMT1A and contribute to finding treatments for other demyelinating CMT types.
CMTA’s commitment to supporting cutting-edge research like this is a crucial step towards finding effective treatments for CMT. Sign up for CMTA eNews to stay current on this and all other CMT research and CMTA news.
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Published On: October 29, 2024