Scientists report studies showing a novel drug repairs myelin and restores nervous system function in a model of multiple sclerosis

et al; was published in the December 21, 2024 edition of Acta Neuropathologica Communications.

IRX4204 selectively activates the retinoid X receptor (RXR). Given compelling links between RXR activation and increased myelin repair, the collaborating scientists employed IRX4204 to investigate the impact of RXR agonism on functional recovery in mice subjected to experimental autoimmune encephalomyelitis (EAE). EAE is a commonly used model of neuroinflammation in human MS. Machine learning was used to obtain highly sensitive and reliable measurements of hindleg movements for mice walking on a treadmill. IRX4204 not only blocked progressive loss of knee and ankle movements, but also reversed joint movement impairments in EAE mice. Biochemical, transcriptional and histological measurements in spinal cord supported that these gait improvements reflected increased axon survival, remyelination, and reduced inflammation. Using microglia, astrocytes and oligodendrocyte progenitor cells, additional data was obtained suggesting that IRX4204 acts on multiple glial subtypes to orchestrate myelin repair.

George S. Robertson, Ph.D. is the lead scientist for these studies. He is a Professor in the Departments of Psychiatry and Pharmacology at Dalhousie University. His laboratory is located in the Brain Repair Centre at Dalhousie University. Dr. Robertson stated “These results inform the discovery of restorative neural therapeutics for MS by demonstrating that selective RXR agonism is sufficient for effective myelin repair. Moreover, our findings support the therapeutic potential of IRX4204 to promote functional neurologic recovery in MS, a long-sought therapeutic objective in the MS research community.”  

Martin E. Sanders, M.D., Chief Executive Officer of Io Therapeutics stated “The ability of IRX4204 to inhibit and functionally repair brain demyelination, while also inhibiting microglia-mediated brain inflammation in an MS model opens opportunities for potentially reparative treatment of brain damage in other types of neurologic conditions in which demyelination and microglial inflammation are demonstrated to play pathologic roles. Examples of such conditions include normal aging-related neurodegeneration, Alzheimer's disease, Parkinson's disease, traumatic injury, stroke, and schizophrenia. The company is planning to develop IRX4204 for multiple nervous system indications.”

About Io Therapeutics: Io Therapeutics, Inc. is a privately held company headquartered in Spring, Texas. More information on Io Therapeutics and its product development programs is available on the company's web site: www.io-therapeutics.com.

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