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    Therapeutic advances in glaucoma focus on retinal ganglion cell pathology

    Strategies for neuroprotection, neuroregeneration, neuroenhancement show promise

    Take-home message: Findings from basic science research elucidating the mechanisms of retinal ganglion cell axon degeneration, regeneration, and death have identified new therapeutic targets for glaucoma that are being investigated in preclinical and clinical studies.


    Stanford, CA—Growing understanding of the pathophysiology of glaucoma is opening the way to the development of strategies for neuroprotection, neuroregeneration, and neuroenhancement.

    While the research is still at an early stage, the progress being made is encouraging, according to Jeffrey L. Goldberg, MD, PhD.

    “Based on animal models, we have every reason to think that retinal ganglion cell (RGC) death occurs as a relatively late event in glaucoma pathophysiology, and that points to a window of opportunity for interventions to protect the RGCs, enhance their function, or enhance regrowth of their axons to targets in the brain,” said Dr. Goldberg, professor of ophthalmology and chairman, Byers Eye Institute, Stanford University, Stanford, CA.

    “In humans, it will be hard to distinguish whether a particular modality is providing neuroprotection, neuroenhancement, or neuroregeneration,” he said. “However, a treatment with any of these activities would be a major step forward.”

    Regenerative strategies aim to overcome the fact that once the axon of a RGC is injured in the optic nerve, it does not regrow to re-establish the connection between the eye and the brain. Based on understanding of the molecular basis for why RGC axons stop growing and of the genes involved, various strategies have been tested in animal models with promising results. Those studies have been conducted with agents that block inhibitory pathways and with neurotrophic factors that might enhance intrinsic growth ability, which seems to be retained but decreases sharply around birth.

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