Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E

Plain-language summary

Charcot-Marie-Tooth disease (CMT) is a hereditary peripheral nerve disease that causes causes muscle atrophy, muscle weakness, sensory loss, balance disorder, gait disorder, blindness, hearing disorder, breathing disorder, vocal cord paralysis, foot deformity, scoliosis, and reflex dysfunction, More than 140 types of genes causing this disease are known. Charcot-Marie-Tooth (CMT) 1E, the target disease of this study, shows very severe symptoms compared to other Charcot-Marie-Tooth types. In cases of early onset, especially in children under 5 years of age, almost all patients are unable to walk without a wheelchair and have severe illness. Symptoms include scoliosis, breathing problems, vocal cord paralysis, foot deformity, loss of sensation and reflex function. Additionally, more than 40% of Charcot-Marie-Tooth (CMT) 1E patients have hearing loss and become unable to live without hearing aids. Although this disease is very disabling, there is still no approved treatment. To date, there is a lack of practical treatment or treatment support methods that can change the progression of hereditary motor and sensory neuropathy, so the focus is on pain control, use of assistive devices, and rehabilitation treatment, but the treatment effect is almost non-existent. This study is conducted for the purpose of confirming the safety and exploratory treatment effect by administering EN001, an allogeneic umbilical cord-derived mesenchymal stem cell, once intravenously to patients with Charcot-Marie-Tooth (CMT) 1E. EN001 is an allogeneic (alien-derived) umbilical cord-derived mesenchymal stem cell, and a phase 1 clinical trial of single intravenous administration was completed in 9 Charcot-Marie-Tooth (CMT) type 1A patients. Among the four adverse reactions that occurred in the participating research subjects, there were no adverse drug reactions related to EN001, and all four cases were mild and recovered. No serious adverse drug reactions or infusion reactions were observed in any study subjects, so this is a safe stem cell treatment. Through efficacy tests and non-clinical tests, the effectiveness of improving behavior and increasing nerve and motor conduction speeds when administering the test drug to animal models of muscle disease was confirmed, so it is expected that this study can stabilize the disease progression in patients, and it will contribute to improving the quality of life and further promoting public health and welfare.

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