The CMT Research Foundation (CMTRF) is closing its September CMT Action Month program with a virtual convention, a $ 10 million research campaign and a partnership with the Muscular Dystrophy Association – all aimed at stimulating collaborations and research to find a cure for Charcot- Marie-Tooth’s disease.
According to a press release shared with Charcot-Marie-Tooth News, CMTRF’s first global CMT research convention, to be held virtually September 24-25, brings together researchers, pharmaceutical companies, government agencies, and patients to discuss, foster collaborations, and streamline efforts to accelerate development of therapies for CMT.
Today’s agenda focuses on sharing the latest advances in therapy, mechanisms and models of disease, and delivering therapies to peripheral nerves – which control movement and sensation in members – among other topics. The full program is available here.
James M. Wilson, MD, PhD, of the University of Pennsylvania, a leader in the development of gene therapies for rare diseases, will deliver the keynote address.
On Saturday, the convention welcomes the CMT patient community, which will be able to hear from researchers and learn about recent developments in CMT clinical trials. To register, go here. Arthur T. Suckow, PhD, CEO of DTx Pharma, will be the keynote speaker for the day.
“The CMT research convention is designed to facilitate greater sharing of ideas, identify areas of need and opportunity for high impact collaborative research projects and inform patients of our remarkable progress in just three years”, said Keith N. Fargo, PhD, Chief Scientific Officer of the CMTRF.
A $ 10 million campaign, to be launched during the convention, has been designed to stimulate research into effective treatments, including gene therapies, for CMT1A, the most common form of the disease.
CMT1A is caused by having an extra copy of the PMP22 gene, which carries the instructions for the PMP22 protein. This protein is an essential component of the myelin sheath, the fatty coating surrounding nerve fibers that improves electrical signals between nerve cells. An excess of PMP22 protein results in the loss of the myelin sheath.
Gene therapies which can stop the excessive expression (activity) of PMP22 have been proposed as a potential strategy for patients with CMT1A with long-term benefits.
“We are at a critical point,” said Peter deSilva, CMTRF board member and campaign chair. “We understand the disease better than ever. It comes down to just one gene. We need to reduce the expression of this gene. “Gene therapies are coming online quickly and new genetic testing capabilities are available.
“Pharmaceutical companies are now interested and keen to develop treatments for CMT1A and companies who are discovering CMT, in partnership with the CMT Research Foundation, have real power to catalyze and accelerate progress. It’s very simple: more capital means more projects with high potential, and that means more chances of processing. We are ready to go bankrupt, ”he added.
Closing September, the CMTRF announced a partnership with the Muscular Dystrophy Association (MDA), the largest and best-known nonprofit for neuromuscular diseases.
The goal of the collaboration is to leverage the scientific expertise of both organizations to promote the therapeutic development of CMT.
“With the MDA, we hope to co-finance transformation projects and join forces to help the [U.S. Food and Drug Administration] better understand CMT and the urgency to commercialize potentially effective treatments, ”said Susan Ruediger, co-founder and CEO of CMTRF. “The CMT scientific community is growing rapidly. We know that real progress is happening in environments that foster open collaboration and the sharing of science. “
During CMT Awareness Month throughout September, the CMTRF encourages the CMT community to start personal fundraisers and raise awareness.