gene therapy

# Forecasting Glaucoma Vision Restoration: 5-, 10-, and 20-Year Outlook **Glaucoma** causes progressive loss of the **retinal ganglion cells (RGCs)** that send visual signals from the eye to the brain. Today’s treatments (medications, lasers or surgery) only **lower eye pressure**, which can slow vision loss but **cannot restore lost nerve cells** ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC9697907/#:~:text=Glaucoma%20is%20a%20neurodegenerative%20disease,studies%20have%20revealed%20effective%20neuroprotection)). In fact, as one recent review notes, “controlling [eye pressure] in certain patients can be futile in slowing disease progression” ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC9697907/#:~:text=Glaucoma%20is%20a%20neurodegenerative%20disease,studies%20have%20revealed%20effective%20neuroprotection)). New research is focused on three approaches: **neurorescue** to save or boost surviving RGCs; **bioelectronic/cortical augmentation** to bypass the damage; and true **regeneration or replacement** of damaged cells. These have very different timelines. Below, we explain what current trials and regulatory paths suggest for each category, using optimistic, base-case, and conservative scenarios. ## Short-Term Outlook (Months–Years): Neurorescue and Neuroenhancement In the next few years, the emphasis will be on **neuroprotection/neuroenhancement** – therapies that aim to **preserve or slightly improve the function of existing RGCs** rather than re-grow them. Studies have identified factors (like neurotrophins or gene signals) that help damaged RGCs survive. For example, **gene therapy** in mice has shown dramatic RGC protection: one Harvard team used three Yamanaka reprogramming factors in mice with glaucoma, and found that injured optic nerves regenerated and vision improved ([www.brightfocus.org](https://www.brightfocus.org/news/a-revolutionary-approach-to-restoring-vision-loss-from-glaucoma/#:~:text=The%20treatment%20turned%20out%20to,related%20vision%20loss)). This proof-of-concept is exciting, but still very early (in mice) and far from a human treatment. More clinically, several early human trials are underway. For instance, a Phase-1 trial used **eye drops containing nerve growth factor (rhNGF)** in glaucoma patients ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC8821405/#:~:text=burden%20mainly%20eliciting%20periocular%20ache,world)). The drops were safe and well-tolerated, but the small trial did **not show a statistically significant vision improvement** over placebo (though there were hints of benefit) ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC8821405/#:~:text=burden%20mainly%20eliciting%20periocular%20ache,world)). In other words, no rescue drug has cleared trials yet. Reviews agree that most neuroprotective strategies (drugs, supplements or cells) that work in animals have **“resulted in approved therapy [for glaucoma] clinically” o

Forecasting Glaucoma Vision Restoration: 5-, 10-, and 20-Year Outlook

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