Age-related macular degeneration affects approximately 20 million adults in the United States, with over 90% presenting the dry form – a progressive condition characterized by retinal pigment epithelial cell dysfunction and death. Until now, therapeutic interventions have been limited to slowing disease progression, with no options for reversing established vision loss.
A phase 1/2a clinical trial published in Cell Stem Cell demonstrates a different approach. Researchers at the University of Michigan transplanted adult-derived retinal pigment epithelial stem cells, sourced from postmortem eye bank tissue, into six patients with advanced dry AMD. These lineage-restricted cells possess the specific capacity to differentiate into the retinal pigment epithelial cells that degenerate in AMD pathology.
Image: ScienceDaily / Shutterstock
The initial cohort received 50,000 cells via surgical transplantation. Safety endpoints were met with no serious adverse events, including inflammation or tumorigenesis. Notably, treated eyes showed functional improvement, with participants gaining an average of 21 letters on standard visual acuity assessments at one-year follow-up – a clinically significant outcome in a population with severe baseline impairment.
According to principal investigator Dr. Rajesh Rao, “We were surprised by the magnitude of vision gain in the most severely affected patients who received the adult stem cell-derived RPE transplants. This level of vision gain has not been seen in this group of patients with advanced dry AMD”. The trial is now evaluating dose-escalation cohorts at 150,000 and 250,000 cells, positioning this cell-replacement strategy as a potential regenerative therapeutic for previously irreversible retinal degeneration.
Research article:
Lambda Biologics’ Oncology Solutions: Patient-derived cancer organoid-based drug evaluation service
Gastric Cancer Organoid | Breast Cancer Organoid | Hepatocarcinoma Cancer Organoid | Pancreatic Cancer Organoid
