Researchers have identified a new therapeutic target for glaucoma which restored nearly 70% of lost vision in animal models.
The Chinese University of Hong Kong (CUHK) researchers said their finding raised hopes for a future treatment that could protect and potentially restore vision in people with the disease.
Findings were published in Advanced Science.
The discovery centres on the growth hormone-releasing hormone receptor (GHRHR), a protein that researchers found plays a key role in the degeneration and recovery of retinal ganglion cells (RGCs) – the nerve cells responsible for transmitting visual information from the eye to the brain.
Glaucoma affects more than 80 million people worldwide and is the second leading cause of blindness globally. Current treatments focus largely on lowering intraocular pressure through medications, laser procedures, or surgery. While these approaches can slow disease progression, they do not repair vision that has already been lost, and many patients continue to experience progressive visual decline despite treatment.
To investigate new therapeutic options, researchers from CU Medicine’s Department of Ophthalmology and Visual Sciences developed several mouse models designed to mimic different forms of glaucoma, including chronic ocular hypertension, acute elevation of intraocular pressure, and optic nerve injury.
Across these models, the team identified GHRHR as a critical regulator of retinal ganglion cell degeneration and recovery.
When the receptor was inhibited, the researchers observed significant neuroprotective effects. Mice treated with GHRHR inhibitors demonstrated marked improvements in visual function, including recovery of nearly 70% of normal light-avoidance behaviour, a commonly used measure of vision in animal studies.
The treatment also increased retinal ganglion cell survival by almost 50%. Researchers found that blocking GHRHR reduced ferroptosis, a form of cell death caused by excessive iron accumulation that has increasingly been implicated in neurodegenerative diseases.
In addition, animals with simulated glaucoma that had lost about 80% of their visual signal transmission capacity regained function equivalent to almost 50% of normal levels within five days of treatment.
Importantly, the researchers reported no structural abnormalities or pathological changes in the retina following GHRHR inhibition, suggesting a favourable safety profile in the preclinical models.
Dr Poemen Chan, Associate Professor (Clinical) in the Department of Ophthalmology and Visual Sciences at CU Medicine, said the findings point to a potentially transformative approach to glaucoma management.
“We have discovered one possible key to restoring vision in glaucoma, as validated across multiple animal models,” Dr Chan said in a media release on 22 June 2026.
“By precisely suppressing harmful substances, GHRHR inhibition not only preserves optic nerve structure but also restores impaired visual function caused by the disease, essentially addressing both the cause and the symptoms.”
Professor Clement Tham, chairman of the Department of Ophthalmology and Visual Sciences at CU Medicine, said the discovery could have implications beyond glaucoma.
Corresponding author Dr Chu Wai-kit said the work provides strong preclinical evidence for a more targeted treatment strategy.
“The discovery provides robust pre-clinical evidence for more precise and effective treatment of primary glaucoma,” he said.
“We aim to translate this innovative approach into clinical applications within five to seven years and will further investigate its potential in high-risk groups such as those with high myopia, offering new hope to more patients with glaucoma.”
Professor Clement Tham, chairman of the Department of Ophthalmology and Visual Sciences at CU Medicine, said the discovery could have implications beyond glaucoma.
“With an ageing population, society is facing a mounting medical and social burden from glaucoma,” Prof Tham said.
“We will continue to advance this innovative neuroprotection strategy, which can potentially transform the treatment of glaucoma and other optic nerve degenerative diseases, and redefine our strategies for blinding diseases.”
