Awareness Month Closing With CMT Research Convention, $10M Funding Campaign

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by Patricia Inacio PhD |

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The CMT Research Foundation (CMTRF) is closing out 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 boosting collaborations and research to find a cure for Charcot-Marie-Tooth disease.

According to a press release shared with Charcot-Marie-Tooth News, the CMTRF’s first Global CMT Research Convention, taking place virtually Sept. 24–25, is bringing together researchers, pharmaceutical companies, government agencies, and patients to discuss, foster collaborations, and streamline efforts to accelerate the development of therapies for CMT.

Today’s agenda focuses on sharing the latest advances in therapeutics, mechanisms and models of disease, and delivery of therapeutics to peripheral nerves — which control movement and sensation in the limbs — among other topics. The full program is available here.

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James M. Wilson, MD, PhD, from the University of Pennsylvania, a leader in gene therapy development for rare diseases, will give the keynote address.

On Saturday, the convention is welcoming the CMT patient community, who will be able to hear from the researchers and learn about the recent developments of CMT clinical trials. To register, go here. Arthur T. Suckow, PhD, CEO of DTx Pharma, will be the keynote speaker on this 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 to update patients on 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, was designed to boost research into effective treatments, including gene therapies, for CMT1A, the most prevalent 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 a critical component of the myelin sheath, the fatty coating surrounding nerve fibers that enhances electrical signals between nerve cells. Excess PMP22 protein leads to loss of the myelin sheath.

Gene therapies that can halt the excessive expression (activity) of PMP22 have been proposed as a potential strategy for CMT1A patients with long-term benefits.

“We’re at a critical point,” said Peter deSilva, CMTRF board member and the campaign’s chair. “We understand the disease better than we ever have. It comes down to a single gene. We need to reduce the expression of that one gene. “Gene therapies are rapidly coming online, and new genetic testing capabilities are available.

“Pharmaceutical companies are now interested and eager to develop treatments for CMT1A and companies new to CMT, in partnership with the CMT Research Foundation, have real power to catalyze and accelerate progress. It’s very simple: More capital means more high-potential projects, and that means more chances for a treatment. We are all-in to put ourselves out of business,” he added.

Closing September, the CMTRF has announced a partnership with the Muscular Dystrophy Association (MDA), the largest and most well-known nonprofit for neuromuscular diseases.

The goal of the collaboration is to leverage scientific expertise from both organizations to help foster therapeutic development in CMT.

“With the MDA we hope to co-fund transformational projects and join forces to help the [U.S. Food and Drug Administration] better understand CMT and the urgency to get potentially successful treatments to market,” said Susan Ruediger, co-founder and CEO of the CMTRF. “The CMT scientific community is growing rapidly. We know that real progress happens in environments that foster open collaboration and sharing of science.”

During CMT awareness month throughout September, the CMTRF is encouraging the CMT community to launch personal fundraisers and raise awareness.