Charcot-Marie-Tooth disease was identified in 1886. Yet, for the next hundred and twenty or so years there was no organized effort to help find an effective treatment, or better yet, a cure even though CMT affects one in 2,500 people (about the same prevalence as multiple sclerosis) including 150,000 Americans and nearly 3 million people around the world.
I am one of them.
In 2006, as part of the patient advocacy group CMTA, I recognized that the traditional methods of finding a treatment or cure for CMT, were not yielding results. There needed to be better communication and coordination between researchers and pharma companies so that knowledge was more quickly shared and there was less duplication of efforts. We brought together all stakeholders including CMT patients, CMT experts, CMT clinicians, and one pharma representative and challenged ourselves to create a program with accountability, collaboration and real-time communication of CMT research and full transparency, in exchange for full funding of identified projects. The result of this work was the Strategy To Accelerate Research (STAR). It was an important step and is still at the foundation of how CMTA administers its research funding.
Today, I believe that technology, new research tools and innovative funding mechanisms can accelerate the pace of progress even more, so we have created the CMT Research Foundation. CMTRF is exclusively focused on funding the discovery and development of treatments and cures for CMT. Our approach again combines investment in the most promising research with a commitment to accountability, collaboration and transparency. Our scientific advisors evaluate a wide range of projects for funding and when companies have promising research, we match them with disease experts to streamline and expedite development. With academic projects, we help match researchers with experts in drug development.
As a partner and not just a source of funds, CMT Research Foundation has adapted elements of the venture capital model that assure if the programs we support come to market and generate a profit, that we share in those proceeds as a way to self-fund additional research. The return on our direct investments may include revenue sharing from licensing agreements or commercialization of the therapeutic, or less commonly, sales of preferred stock.
As a result of STAR and the very important work of organizations like CMTA, the Hereditary Neuropathy Foundation, the Muscular Dystrophy Association among others, more has been accomplished in the past five years toward finding effective treatments and a possible cure than ever before. With the creation of the CMT Research Foundation, I see a light at the end of the tunnel. Robust efforts being made by researchers on CMT1A, 1X, 2A, 2E and 4, in partnership with biotech and pharmaceutical companies should give the CMT community hope!
You have my commitment and that of the CMT Research Foundation to drive research forward at an unprecedented pace. I look forward to sharing news and updates about our research efforts as they happen.
Patrick Livney, Co Founder and Board Chair
CMT Research Foundation
At 59, may not help me much, but for future generations, wonderful. Just coming to grips with this CMT. and having hard time with it. Good to read about your dedication. Thanks
Thank you Patrick for this information. I too have it and was misdiagnosed as a child because no treatment they didn’t teach about in Med school ☹️ I was diagnosed with “undiagnosed polio” in elementary school. I was having trouble walking and fell down a lot as a younger child because I am before the polio vaccine came out and it sometimes affected the feet. I was diagnosed with CMT until I was 49 and had chemo and the neuropathy didn’t go away when out of my system which it should have. I was an oncology nurse and knew that. Since someone had found the gene my doctor must have heard about it because when all the tests for the other diseases came back negative he did a genetic test.