2021 has been a banner year for progress in CMT drug development, much of which has been possible because of your partnership with the CMT Research Foundation – thank you! Despite the challenges presented by a global pandemic, we can be proud of all that we were able to accomplish. Together we are making an impact on the pace of efforts to develop treatments for CMT.
Read on to see just some of what you made possible in 2021 alone.
Started Four New Drug Development Projects
CMT1A: Georgia Tech and Emory University. Led by Dr. James Dahlman, this project aims to develop new gene therapy delivery tools to effectively reach the peripheral nervous system. The project utilizes lipid nanoparticles (LNPs), which are FDA approved for another genetic condition and now widely utilized to deliver the most common Covid-19 vaccines. This project will identify LNPs that target Schwann cells and other cells found in the peripheral nerves, making his research potentially relevant to many types of CMT. [Read more]
CMT1X: University of Illinois Chicago. Led by Dr. Charles Abrams, this project is testing inosine as a potential therapeutic for CMT1X. Known to be deficient in people with CMT1X, inosine has anti-inflammatory properties and may prevent, slow, or even reverse symptoms in people with CMT1X. [Read more]
CMT1X: Loyola University Chicago. Led by Dr. Kelly Langert, this project aims to develop and test a tool to deliver compounds to the peripheral nervous system. Taking advantage of the nerve inflammation seen in CMT1X, this “Trojan horse” is designed to trick the inflamed nerves into allowing it entry, where it can release a payload of therapeutic molecules where they are needed most. [Read more]
CMT4B1: Ospedale San Rafaelle and AcuraStem. Led by Dr. Alessandra Bolino, this project is testing AcuraStem’s next generation PIKfyve inhibitor to treat CMT4B1. Designed to be more stable in the body than earlier PIKfyve inhibitors, this therapeutic candidate may restore nerve function in CMT4B1 and other subtypes that involve myelin outfoldings. [Read more]
With these new projects added, we are pleased to report that we now have programs in all four major subtypes (CMT1A, CMT1B, CMT2A, and CMTX), and several of the rarer subtypes as well.
Completed Three Drug Development Projects
CMT1A: DTx Pharma. Led by Artie Suckow and Rafaella Gesuete, this project identified a novel genetic medicine to treat CMT1A. Based in part on the strength of this program, DTx has now attracted over $100M in outside investments to drive their gene therapy program forward and is now focused on preparing for clinical trials. [Read more] [Watch video]
CMT1A: Cyprus Institute of Neurology and Genetics (CING), in collaboration with Nationwide Children’s Hospital (NCH). Led by Dr. Kleopas Kleopa of CING and Dr. Scott Q. Harper of NCH, this project created a viral vector-based genetic medicine to treat CMT1A. This therapeutic is now in the process of being licensed by Armatus Bio for further research and potential clinical trials. [Read more] [Watch video]
CMT2A: AcuraStem. In this drug screen project, scientists at AcuraStem developed a screening platform utilizing cells derived from a person with CMT1B. After screening several thousand compounds—many of them already approved drugs—the scientists have identified 11 potential therapeutics for CMT2A. [Watch video]
Achieved Two Key Milestones in Drug Development
CMT1B: The Jackson Laboratories (JAX). Designed by the CMT Research Foundation Scientific Advisory Board in collaboration with the scientists at JAX, this project created three new genetic models for CMT1B research. Designed to speed drug discovery, each has a humanized version of MPZ, the gene that is implicated in CMT1B. The models include one with a healthy gene, one with an early onset mutation, and one with a late onset mutation. [Read more] [Watch video]
CMT2E: University of Missouri. Led by Dr. Chris Lorson and Dr. Michael Garcia, this project is developing a “knock down and replace” genetic medicine that is designed to work on anyone with CMT2E, regardless of their specific mutation. In the first stage, completed this year, the researchers identified three new sequences that knock down expression of the culprit gene. [Read more]
Launched Two Key Initiatives to Stimulate Drug Development
Endgame: In September, we launched Endgame, a 3-year campaign to raise $10M to support CMT1A research. While we are excited about the new possibilities this campaign is creating in CMT1A research, we also know that this critical work will have benefits for people with all subtypes of CMT. To date, thanks to your enthusiasm and generosity, we have already raised over $4.6M toward the $10M goal.
Global CMT Research Convention: 2021 marked the first annual Global CMT Research Convention. Open to all and covering two full days, this virtual event drew more than 160 scientists from 20 countries, representing academic institutions, pharma, and biotech companies. The second day of the convention, programmed for patients and families, drew nearly 300 attendees from 34 countries. [View the recordings]
Started Two New Groups of Volunteers
Young Professionals Board: The Young Professionals Board is a group of highly motivated young professionals who give their time and talents to support the CMTRF’s key initiatives. [Read more]
CMT Research Foundation Ambassadors: The Ambassadors span the globe building awareness and funding for the CMT Research Foundation. [Read more]
Welcomed a New Director to the Board