Researchers from Osaka University have achieved a significant breakthrough in regenerative medicine by successfully restoring human vision using induced pluripotent stem cell (iPSC)-derived corneal epithelium.
This first-in-human trial addressed limbal stem cell deficiency (LSCD), a severe ocular condition causing vision impairment due to the loss of functioning stem cells at the cornea’s edge. The study involved four LSCD patients who received transplants of iPSC-derived corneal epithelial sheets (iCEPS). The surgeries were performed without human leukocyte antigen matching, and patients were monitored for two years.
Results showed significant vision improvements in all patients, with three experiencing less severe disease stages. Quality-of-life assessments also improved for most participants. The iCEPS were cultivated using a method mimicking natural eye development, ensuring graft integrity and reducing immunogenicity. This approach potentially eliminates the need for extensive immunosuppression typically required in traditional grafts. Patients receiving low-dose cyclosporine showed more pronounced benefits, suggesting its importance in preventing subclinical rejection.
This breakthrough offers a promising alternative to traditional treatments, which often involve grafts from the patient’s healthy eye or donors, carrying risks of immunological rejection or requiring removal of healthy tissue. The success of this trial paves the way for larger multicenter clinical trials to further validate the findings and explore broader applications of iCEPS transplantation in treating LSCD and restoring vision.
