A new stem cell treatment for a previously untreatable rare genetic eye condition – aniridia-related keratopathy (ARK) – has improved vision for patients in a first-in-human clinical trial at Moorfields Eye Hospital and University College London (UCL).
ARK is a progressive ocular surface disorder associated with limbal stem cell deficiency and limited effective treatment options. It leads to conjunctival tissue growing over the cornea, turning the eye opaque and forming a thickening layer that gradually results in profound vision loss. It is mostly an inherited genetic eye disease.
Researchers at Moorfields and UCL performed a Real Architecture for 3D Tissues-Ocular Surface (RAFT-OS) transplant in nine trial participants.1
In JAMA Ophthalmology, they reported RAFT-OS transplant was feasible and associated with early ocular surface improvement, partly sustained through 12 months, without substantial safety concerns.2
… the trial was “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”
Professor Sajjad Ahmad, from both Moorfields Eye Hospital and the UCL Institute of Ophthalmology, said the trial was “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.”2
Nine adults with congenital aniridia with advanced ARK underwent RAFT-OS transplant. They started the trial with an average ocular surface score of 9.4 in the treated eye, and 8.4 in the untreated. The treated eyes improved to 5.9 after three months (6.7 after 12), while the untreated eyes had changed very little.
Importantly, when measuring best corrected visual acuity, there was a mean change of −0.47 logMAR – or almost five lines of visual acuity – in the treated eyes after 12 months while there was essentially no average change in the fellow, untreated eyes.
The researchers said additional controlled studies with longer follow-up are needed to define safety and clinical effects.
References
- University College London, Stem Cell Therapy Restores Sight for Untreatable Condition, Mirage News, 19 Aug 2026, available at: miragenews.com/stem-cell-therapy-restores-sight-for-1729158 [accessed Aug 2026].
- Kaye AE, Morgan L, Ahmad S, et al. Combined limbal epithelial and stromal cell transplant for aniridia-related keratopathy: a nonrandomized clinical trial. JAMA Ophthalmol. Published online July 23, 2026. doi: 10.1001/jamaophthalmol.2026.2701.
