Stem Cell Treatment Feasible for Aniridia-Related Keratopathy

TUESDAY, Sept. 8, 2026 (HealthDay News) — Real Architecture for 3D Tissues-Ocular Surface (RAFT-OS), a tissue-engineered collagen scaffold incorporating allogeneic limbal epithelial stem cells and stromal keratocytes, is feasible and associated with early ocular surface improvement in adults with advanced aniridia-related keratopathy (ARK), according to a study published online Aug. 18 in JAMA Ophthalmology.

Abigail Eve Kaye, M.B.Ch.B., Ph.D., from the Moorfields Eye Hospital NHS Foundation Trust in London, and colleagues conducted a single-arm, open-label, nonrandomized clinical trial to examine the safety, feasibility, and clinical outcomes of RAFT-OS in nine adults with advanced ARK; untreated fellow eyes were natural history comparators.

One early serious adverse event prompted amendment of the manufacturing protocol; there were no further major RAFT-OS-related safety events. The researchers found that persistent epithelial defects developed in two participants. The mean Ocular Surface Score (OSS) was 9.4 at baseline in treated eyes, and 5.9 and 6.7 at three and 12 months, respectively. In untreated eyes, mean OSS was 8.4, 8.4, and 8.0 at baseline, three, and 12 months, respectively. At baseline and 12 months, the mean treated eye best-corrected visual acuity (BCVA) was 2.23 and 1.77 logMAR (Snellen equivalent, <20/1,600 and 20/1,280, respectively); mean fellow eye BCVA was 1.47 and 1.44 logMAR at baseline and 12 months (Snellen equivalent, 20/640 and 20/640, respectively).

“This is the first-ever optical use of a collagen scaffold containing two types of stem cells and has proved effective for a previously untreatable progressive condition leading to sight loss,” coauthor Sajjad Ahmad, M.B.B.S., Ph.D., also from Moorfields Eye Hospital NHS Foundation Trust, said in a statement.

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