From time to time the CMT Research Foundation will examine the impediments to finding a cure for CMT.
As the Chairman of a nonprofit foundation whose ONLY mission is to fund research that will lead to a cure for Charcot-Marie-Tooth disease (CMT), I am often asked, “How long before you are successful?”
That is a fair question and one that I wish, as a patient and a source of research funding, I could answer with more confidence. There are many efforts aimed at finding solutions for the different types of CMT, with more than half a dozen options being prepared for clinical trials. The momentum is strong and after ten years of persistence, CMT finally has the interest of both small biotech and large pharmaceutical companies.
They see the same landscape I do which makes finding a CMT treatment or cure attractive to them:
- CMT is considered a rare disease by the regulatory agencies, so it qualifies for incentives to make the treatment approval process faster.
- CMT is not that rare. Around 3 million people live with it worldwide, creating a relatively strong market for any treatment options.
- For the most common types of CMT, we understand the genetic cause and how they create neuropathy.
- Over 10,000 patients are registered with the INC patient registry, making the patient registry one of the largest in the rare disease world. This gives research and pharma companies access to macro natural history studies.
- There are more than 300 researchers dedicated to answering the most important questions about CMT.
The biggest single contributor to CMT research is the Federal government’s National Institutes of Health (NIH), which funds around $12 million in CMT research out of a $39.2 billion annual medical research budget.
At the same time, the U.S. government spends $112 million annually on Multiple Sclerosis (MS) although more people around the world have CMT than MS. MS has seventeen treatment options; CMT has zero. CMT is three times bigger than ALS, yet in 2018, NIH spent $83 million on ALS. Relative to the number of people affected, CMT doesn’t get ample support from our leaders in Congress who determine the allocation of funding for each disease.
While CMT is drastically underfunded by the NIH, leaders like Dr. Chris Austin, the Director of the National Center for Advancing Translational Research, is nevertheless an advocate for CMT research and is energized by the work at the CMTRF. Our recent visits to the NIH and several meetings with Dr. Austin have led to strategies aimed at overcoming the biggest barriers in CMT research. We’ve prioritized projects and are gathering information and proposals from the most accomplished researchers in the field. We are pleased to collaborate with their leaders in gene therapy, cellular biology and translational medicine. These efforts will expedite the development of treatments for CMT.
The CMT Research Foundation has already committed almost $800,000 to innovative projects and are actively reviewing more. To learn more about our currently funded projects, please visit https://cmtrf.org/research-we-fund/. We are actively engaging researchers and industry to find and fund new projects and will announce new partnerships in the coming weeks.
Your support plays a vital role in improving the lives and health of everyone affected by CMT. Your gift, no matter how large or small matters. An investment in CMT research is transformative when united with expertise, a strong network, a singular strategy and a vision for success. The CMT Research Foundation has these elements, but cannot be successful without your support.