Stem cell-based therapies novel strategy that may substitute conventional corneal transplantation

Limbal biopsies were used to generate CALEC constructs, and final grafts were evaluated by noninvasive scanning microscopy and tested for viability and sterility. Cultivated cells maintained epithelial cell phenotype with colony-forming and proliferative capacities. Analysis of LSC biomarkers showed preservation of “stemness.”

After preclinical development, a phase 1 clinical trial enrolled five patients with unilateral LSC deficiency. Four of these patients received CALEC transplants, establishing preliminary feasibility. Clinical case histories are reported, with no primary safety events. On the basis of these results, a second recruitment phase of the trial was opened to provide longer term safety and efficacy data on more patients.

In summary, the initial phase of this trial has established feasibility of the product manufacturing method with no immediate safety concerns, enabling a second recruitment phase to provide longer term safety and efficacy data on more patients. Use of the small biopsy carries low risk of inducing LSCD in the donor eye, as seen in one example showing the repeatability of the CALEC procedure.

Reference: urkunas UV, et al "Cultivated autologous limbal epithelial cell (CALEC) transplantation: Development of manufacturing process and clinical evaluation of feasibility and safety" Sci Adv 2023; DOI: 10.1126/sciadv.adg6470.