Researchers led by Osaka University in Japan have conducted the first human trial using induced pluripotent stem-cell-derived corneal epithelium to treat limbal stem cell deficiency, offering a potential new avenue for restoring vision.
Limbal stem cell deficiency (LSCD) is a severe ocular condition where the loss of functioning adult stem cells at the cornea’s edge leads to vision impairment due to the invasion of fibrotic conjunctival tissue over the cornea. Limbal stem cells normally perform repair functions by differentiating into corneal epithelium. Without them, the integrity and transparency of the corneal surface becomes compromised, leading to fibrotic tissue buildup, and ultimately, vision loss.
Traditional treatments often involve grafts from the patient’s healthy eye or donors, but these methods carry risks like immunological rejection, or require the removal of healthy tissue.
In a study titled “Induced pluripotent stem-cell-derived corneal epithelium for transplant surgery: a single-arm, open-label, first-in-human interventional study in Japan,” published in The Lancet, researchers conducted transplants of pluripotent stem cell (iPSC)-derived corneal epithelial sheets (iCEPS) as a potential treatment for LSCD.
Four patients with LSCD participated in the study. After removing any fibrotic tissue, the team transplanted allogeneic iCEPS onto the affected eyes. All surgeries were performed without human leukocyte antigen (HLA) matching. Half the patients received low-dose cyclosporine (typically used to mitigate organ rejection after a transplant), while the other half received no immunosuppressive agents beyond corticosteroids.
Two years of monitoring revealed no severe adverse events. Minor adverse events were managed effectively and without lasting effects.
By Justin Jackson , Medical Xpress
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