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Researchers led by Rajesh C. Rao, M.D., have reported early but promising results from a first-in-human phase 1/2a clinical trial testing adult stem cells as a potential therapy for age-related macular degeneration (AMD). The study, published in Cell Stem Cell, focuses on the dry form of AMD, which accounts for more than 90% of cases and remains without any treatment capable of restoring lost vision. Dry AMD occurs when retinal pigment epithelial (RPE) cells in the central retina become dysfunctional and eventually die, compromising the sharp, colour-rich central vision needed for tasks like reading or recognising faces.
In this trial, scientists used RPE stem cells derived from donated postmortem eye tissue. These adult stem cells are already committed to becoming RPE cells, making them well-suited for replacing the specific cell type lost in AMD. Six patients with advanced dry AMD received a low dose of 50,000 cells delivered through a surgical procedure to the affected eye. The primary aim was to assess safety, and the results were encouraging: no serious inflammation, abnormal cell growth, or tumour formation occurred.
Beyond safety, the researchers observed something unexpected—meaningful improvements in vision. Participants in the low-dose group could read 21 more letters on an eye chart one year after treatment. Their untreated eye did not show the same gains, suggesting the transplant itself drove the improvement. The effect was particularly striking in patients who had been most severely impaired before treatment.
The research team is now monitoring individuals who received higher doses of 150,000 and 250,000 cells. If safety continues to hold, the therapy will advance to later-phase trials. While still early, these findings mark an important step toward regenerative treatments that could one day restore vision for millions living with dry AMD.
