Osaka, Japan – A revolutionary stem cell transplant has significantly improved vision for three patients suffering from severe corneal damage due to limbal stem cell deficiency (LSCD). This first-of-its-kind clinical trial, conducted in Japan, marks a major advancement in stem cell research and offers new hope for millions affected by corneal vision loss.
Two years post-operation, no major safety concerns have arisen, and all three patients’ corneas appear noticeably clearer. LSCD, the disorder affecting these patients, leads to scar tissue buildup on the cornea, impairing vision. While current treatments for LSCD in a single eye involve transplanting healthy corneal tissue from the other eye, patients with damage in both eyes require scarce donor transplants.
Globally, only one in seventy people experiencing corneal vision loss receive a transplant, and grafts often face survival issues, including the risk of rejection.
This trial explores a new approach using induced pluripotent stem cells (iPSCs), which are reprogrammed from human cells and can develop into any cell type. Researchers at Osaka University Hospital used iPSCs derived from healthy blood cells to create corneal epithelial cell sheets (iCEPS). These cell sheets were then transplanted over the damaged cornea after scar tissue removal, followed by a protective contact lens.
Within seven months, all four patients experienced visual improvements. However, by one year, one patient with severe vision loss saw her improvements diminish. The other three patients, including a 44-year-old woman and a 66-year-old man, saw the most significant and lasting improvements. Researchers suspect that patients who saw less improvement may have experienced an immunological response, as no immunosuppressive drugs were administered except steroids.
While previous attempts to restore vision with iPSCs used a patient’s own cells to repair macular degeneration, this trial is the first to address LSCD using cells from healthy donors. The study’s promising results lay the groundwork for further research into the safety and potential of iPSC-based transplants.
“To our knowledge, this is the first time iPSC-derived cell constructs have been transplanted onto patients’ corneas,” stated the team at Osaka University Hospital, who now plan a multicenter clinical trial to expand on these encouraging results.