According to a story from Charcot-Marie-Tooth News, the drug company DTx Pharma has recently obtained critical funding that will go towards the development of new RNA-based treatments for Charcot-Marie-Tooth disease type 1A, a rare disease. This variant is the most widespread form. The funding was acquired through a grant from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health (NIH). These therapies will be designed with a mechanism to lower levels of PMP22 in order to restore cellular function.
About Charcot-Marie-Tooth Disease (CMT)
Charcot-Marie-Tooth disease is a hereditary disorder of the peripheral nervous system. It is most characterized by a progressive loss of touch sensation and muscle tissue in several different parts of the body. The cause of this disease is usually linked to a genetic mutation, but the mutation involved varies depending on the variant of Charcot-Marie-Tooth disease. There are multiple types of Charcot-Marie-Tooth disease, with all types aside from type 2 having a demyelination effect. Type 2 causes damage to the neuronal axon instead. Symptoms include foot drop, muscle wasting (typically in the arms, legs, and hands), painful muscle spasms, loss of sensation in the limbs, scoliosis, trouble speaking, chewing, and swallowing, and tremors. Treatment typically includes therapy and surgery in order to maintain function. There is no cure. The disease can occur early in life or as late as the 30s and 40s. To learn more about Charcot-Marie-Tooth disease, click here.
Earlier studies supported by the CMT Research Foundation have already revealed certain molecules that appear to be effective in bringing down PMP22. These findings were the basis for the company’s application for the grant. The company’s focus has been on the development of short interfering RNA molecules. They bind to the messenger RNA from PMP22. This is intended to halt production of the protein and allow for the restoration of the myelin sheath.
DTx uses a proprietary delivery method called FALCON in order to get the molecules delivered effectively. This has been an issue with RNA based therapies in the past, which can be cleared rapidly by the body. The new funding boost will allow the company to develop its approach more thoroughly and potentially produce a breakthrough therapy for type 1A Charcot-Marie-Tooth disease.