Small molecule offers great therapeutic potential for restoring vision

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Researchers at City University of Hong Kong (CityU) have identified and demonstrated for the first time a therapeutic small molecule, M1, that can restore the visual function in the mammalian central nervous system (CNS), offering hope for patients with optic nerve damage such as glaucoma-related vision loss.

Traumatic injuries to the CNS, including the optic nerve, the brain and the spinal cord, are the leading causes of disability worldwide for which there is no available treatment. M1 stimulates the fusion and motility of mitochondria (the powerhouse of a cell to generate energy) and induces robust axon regeneration by enhancing the intrinsic growth capacity of injured neurons.

Led by Dr Eddie Ma Chi-him, Associate Head and Associate Professor in the Department of Neuroscience and Director of the Laboratory Animal Research Unit at CityU, this research breakthrough heralds a new approach that could address unmet medical needs in accelerating functional recovery within a limited therapeutic time window after CNS injuries.

“Photoreceptors in the eyes [retina] forward visual information to neurons in the retina. To facilitate the recovery of visual function after injury, axons of neurons must regenerate through the optic nerve and relay nerve impulses to visual targets in the brain via the optic nerve for image processing and formation,” said Dr Ma.

“M1 treatment sustains long-distance axon regeneration from the optic chiasm, i.e. midway between the eyes and target brain region, to multiple subcortical visual targets in the brain. Regenerated axons elicit neural activities in target brain regions and restore visual functions after M1 treatment. Our study highlights the potential of a readily available and non-viral therapy for CNS repair.”

The seven-year-long study builds on the team’s previous research on peripheral nerve regeneration using gene therapy.

“This time we have used the small molecule M1 for repairing…

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