The randomised, partially blinded Phase I/II trial is said to be the first of its kind to evaluate the safety and efficacy of using stem cells from donors to create tissue for repairing the damage caused by LSCD.
LSCD affects the stem cell regeneration ability, leading to severe vision loss and chronic pain.
Previous research showed that autologous limbal stem cell transplantation, involving isolation of stem cells from healthy tissue in the patient’s own eye could be used to treat the eye disease.
However, this approach cannot be used when both eyes are affected by LSCD.
During the latest trial, the researchers obtained corneal epithelial stem cells from a deceased donor and cultured them on amniotic membrane, which was considered as the investigational medicinal product (IMP) during the study.
They believed that the trial’s controlled design would quantify the original contribution of stem cells in the IMP.
Scottish National Blood Transfusion Service co-investigator John Campbell said: “We hypothesised that the use of amniotic membrane, topical autologous serum eye drops and systemic immunosuppression may have therapeutic benefit.
“By this highly controlled design it was anticipated that the true contribution of stem cells in the IMP would be quantified.”
Trial participants received corneal epithelial stem cells cultured on amniotic membrane or control amniotic membrane alone. The investigators did not observe any IMP-related adverse reactions after 18 months.
Nine out of the 13 participants experienced improvement in their visual acuity scores, without any statistically significant differences between the IMP and control groups.
In addition, patients who received IMP are said to have achieved significant, sustained improvement in ocular surface score, compared to those in control arm.
University of Edinburgh clinical ophthalmology professor Baljean Dhillon said: “This is the first RCT in this field and sets the standard for multidisciplinary clinical trial design and delivery of cell-based therapeutics in regenerative ophthalmology.”
Data from the research has been published in STEM CELLS Translational Medicine.