Small molecule’s big potential for restoring vision

16 January 2023

Researchers at City University of Hong Kong 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, offering hope for patients with optic nerve damage such as glaucoma-related vision loss.

Traumatic injuries to the central nervous system, 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 mobility of mitochondria (the powerhouse of a cell to generate energy) and induces healthy axon regeneration by enhancing the growth capacity of injured neurons.

This research breakthrough provides a new approach that could address unmet medical needs in accelerating functional recovery within a limited therapeutic time window after injuries to the central nervous system. The team was led by Dr Eddie Ma, associate professor in the Department of Neuroscience and director of the Laboratory Animal Research Unit at City University of Hong Kong, 

“Photoreceptors in the retina transport 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,” Ma said.

“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 research team is developing an animal model for treating glaucoma-related vision loss using M1 and possibly other common eye diseases and vision impairments such as diabetes-related retinopathy, macular degeneration and traumatic optic neuropathy.

“Nerve regeneration and function recovery will help improve the quality of life for patients and reduce the burden on the local community and healthcare systems,” Ma added.

Their research was published in the Proceedings of the National Academy of Sciences.