NDC-1308 is a drug being developed by Endece Neural to repair the myelin sheath of demyelinated axons (nerve fibers), a major cause of neurodegenerative disorders like multiple sclerosis (MS). It is currently in preclinical validation studies, and its development is partly supported by a research grant from the National Multiple Scerosis Society. The therapy is specifically targeted to heal motor neuron damage in people with secondary progressive MS (SPMS), and is intended for use either as a monotherapy or in combination with other disease-modifying agents to slow MS progression.
How NDC-1308 Works
The mechanism of action for NDC-1308, a small molecule, focuses mainly on the remyelination of nerve fibers. Damage to myelin sheaths due to immune responses and inflammatory pathways is a key factor in MS disease pathogenesis. The formulation works on a chain of key regulatory gene pathways responsible for differentiation of oligodendrocyte progenitor cells (OPC) and myelin synthesis. NDC-1308 has been shown in preclinical models to cross the blood-brain barrier effectively, and to exert its effects on motor neurons in the brain and spinal cord, where myelin production is most needed. The molecule works by inducing differentiation of OPCs into mature oligodendrocytes, cells that synthesize and maintain the myelin sheath.
NDC-1308 in Preclinical Trials
Preclinical tests are reported to demonstrate that NDC-1308 induced significant remyelination in several brain regions of mice studied, in which the neurotoxicant cuprizone was used to remove myelin sheath from their axons. In addition, in an experimental autoimmune encephalomyelitis (EAE) mouse model of brain inflammation, NDC-1308 was seen to delay the onset of clinical MS symptoms while preserving neuronal cells, suggesting that it also exerts a neuroprotective effect. Treatment was well-tolerated by the animals, suggesting NDC-1308 can be safely administered. The results of the preclinical trials were presented at the American Academy of Neurology conference in 2016.
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