A revolutionary dual stem cell scaffold has successfully improved vision and eye surface scores in a small first-in-human clinical trial for aniridia-related keratopathy. For individuals suffering from ARK, progressive damage to the eye’s surface gradually clouds the cornea and severely compromises sight.
Conducted jointly at Moorfields Eye Hospital and University College London (UCL), the groundbreaking trial offered new hope for patients with this previously untreatable genetic condition. The promising findings were officially published in the esteemed journal JAMA Ophthalmology.
Understanding Aniridia-Related Keratopathy
Aniridia is a complex eye surface disorder involving limbal stem cells, which reside at the boundary between the sclera and the cornea. Typically triggered by an inherited genetic abnormality impacting eye development, the condition frequently leads to profound vision loss.
ARK is a severe, progressive form of aniridia caused by a mutation in the PAX6 gene, affecting between one in 40,000 and one in 100,000 individuals. When a shortage of limbal stem cells occurs, conjunctival tissue aggressively spreads across the cornea, rendering it opaque and progressively blocking vision.
Innovative Collagen Scaffold and Clinical Results
Researchers at Moorfields and UCL engineered a specialized collagen scaffold designed to deliver two distinct types of stem cells directly to the affected eye. Each of the nine trial participants received the advanced treatment in one eye, while the untreated eye acted as a crucial control.
At the start of the study, the nine patients registered an average ocular surface score of 9.4 in treated eyes, which significantly improved to 5.9 after three months and settled at 6.7 after twelve months. Meanwhile, vision scores on the ETDRS test surged by an average of 24 letters, marking a massive recovery equivalent to nearly five lines on a standard clinical chart.
Paving the Way for Future Treatments
Discussing the breakthrough, “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. We are now looking to take this forward to a larger clinical trial, with a view to this being accepted as an NHS treatment,” stated Professor Sajjad Ahmad, consultant ophthalmic surgeon at Moorfields Eye Hospital.
The vital research was generously supported by funding from the Medical Research Council, Moorfields Eye Charity, and the NIHR Biomedical Research.
