Limbal stem cell deficiency

Limbal stem cell deficiency (LSCD), also known as limbal stem cell failure, is a condition affecting the eye in which the stem cells located at the limbus — the border between the cornea and the white of the eye (sclera) — are damaged, depleted, or dysfunctional. These limbal stem cells are essential for maintaining and regenerating the corneal epithelium, the transparent outer layer of the cornea that is critical for clear vision. When these stem cells are lost or impaired, the corneal surface can no longer renew itself properly, leading to invasion of conjunctival tissue and blood vessels onto the cornea (conjunctivalization), chronic inflammation, recurrent epithelial defects, corneal scarring, and progressive vision loss. LSCD can be unilateral (affecting one eye) or bilateral (affecting both eyes) and may be partial or total. Key symptoms include persistent eye pain, tearing, photophobia (light sensitivity), redness, blurred vision, and recurrent corneal erosions. The condition can be acquired or hereditary. Acquired causes include chemical or thermal burns, contact lens overuse, multiple ocular surgeries, severe infections, and chronic inflammatory conditions such as Stevens-Johnson syndrome or ocular cicatricial pemphigoid. Hereditary causes include aniridia (associated with PAX6 gene mutations), ectodermal dysplasia, and other developmental disorders affecting the ocular surface. Treatment depends on the severity and whether one or both eyes are affected. Mild cases may be managed with preservative-free lubricants, anti-inflammatory medications, and autologous serum eye drops. For more advanced unilateral disease, limbal stem cell transplantation from the healthy fellow eye (conjunctival-limbal autograft) or from a living related or cadaveric donor (allograft) can be performed. Cultivated limbal epithelial transplantation (CLET) and cultivated oral mucosal epithelial transplantation (COMET) are advanced cell-based therapies that have shown promising results. In Europe, Holoclar (ex vivo expanded autologous human corneal epithelial cells containing stem cells) was the first approved stem cell therapy for LSCD caused by burns. Severe bilateral cases may ultimately require keratoprosthesis (artificial cornea) if other treatments fail.

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Common questions about Limbal stem cell deficiency