Limbal stem cell deficiency (LSCD) results from the loss or dysfunction of epithelial stem cells and can seriously impair sight. Autologous limbal stem cell transplantation is effective in treating unilateral and partial bilateral disease, but not total bilateral disease. Researchers recently discovered they could culture cadaveric corneal tissue and use it to manufacture allogeneic ex vivo expanded corneal epithelial stem cells on an amniotic membrane. Knowing this, they then conducted a controlled multicenter study to examine the feasibility, safety and efficacy of allogeneic corneal epithelial stem cells in the treatment of bilateral LSCD.

Sixteen patients were randomized to receive corneal epithelial stem cells cultured on amniotic membrane (AM), investigational medicinal product (IMP) or control AM. The primary endpoints were safety and visual acuity, and the secondary endpoint was composite ocular surface score (OSS).

Of the total patients, 13 completed all assessments. Nine of the 13 had improved visual acuity scores at the end of the trial, with no significant differences between the IMP and control groups, the team said in the study. They note that patients in the IMP group demonstrated significant, sustained improvement in OSS, whereas those in the control group did not.

The study concludes that using allogeneic corneal epithelial stem cells is a safe, feasible approach to treating severe bilateral LSCD and that other studies examining specific etiologies of LSCD will further define the role of this technique in treating severe bilateral disease.