Japan Uses iPSC Cell Sheets for the First Time to Treat Corneal Diseases

On November 7, 2024, researchers from Osaka University published a research paper entitled "Transplantation using Induced pluripotent stem cell-derived corneal epithelial cell tablets: a single-arm, open-label, first-in-human intervention study in Japan" in The internationally renowned medical journal The Lancet.

This clinical study is the first in the world to use corneal epithelial cell tablets (iCEPS) derived from human induced pluripotent stem cells (iPSC) to repair corneas in patients with visual impairment due to limbal stem cell deficiency (LSCD). The results of the study show that this new type of regenerative therapy is safe and helps to improve patients' vision. The research team plans to conduct larger clinical trials to gain a deeper understanding of the treatment's efficacy.

In this study, ophthalmologist Kohji Nishida and his team at Osaka University used induced pluripotent stem cell (iPSC) technology to treat limbal stem cell deficiency (LSCD). The team took blood cells from a healthy donor and reprogrammed them into IPscs, which were then transformed into a thin, transparent sheet of oval corneal epithelial cells.

During the study, the research team recruited two male and two female patients, aged 39 to 72 years, all with binocular limbal stem cell deficiency (LSCD). The team first removed the scar tissue covering one of the patient's eyes, then stitched human IPSC-derived corneal epithelial cell sheets (iCEPS) and fitted the patient with flexible protective contact lenses.

At the 52nd week of follow-up, secondary measures of the clinical trial showed improvement in disease staging, improved corrected distant vision, and reduced corneal opacity in all treated eyes. Although one patient experienced a slight regression in vision improvement over the one-year monitoring period, the improvement was sustained in the remaining three patients.