Experimental Stem Cell Treatment Shows Success in Restoring Corneas
A clinical trial testing an stem cell treatment for blinding cornea injuries has shown promising results.
The expanded phase 1/2 trial, published in Nature Communications[1] found the treatment to be feasible and safe for 14 patients who were followed for 18 months, with a high proportion of complete or partial success.
The treatment, known as cultivated autologous limbal epithelial cells (CALEC), was developed at Mass Eye and Ear, a member of Mass General Brigham. The procedure involves harvesting stem cells from a patient’s healthy eye through a biopsy, expanding them into a cellular tissue graft in a specialized manufacturing process over 2 to 3 weeks, and then transplanting the graft into the damaged cornea.
“Our first trial in four patients showed that CALEC was safe and the treatment was possible,” principal investigator Ula Jurkunas, MD, associate director of the Cornea Service at Mass Eye and Ear and professor of Ophthalmology at Harvard Medical School, said in a Mass Eye and Ear article. “Now we have this new data supporting that CALEC is more than 90% effective at restoring the cornea’s surface, which makes a meaningful difference in individuals with cornea damage that was considered untreatable.”
The study showed that CALEC completely restored the cornea in 50% of participants at their 3-month follow-up, with the success rate rising to 79% and 77% at 12 and 18 months, respectively. With two participants achieving partial success at both 12 and 18 months, the overall success rate reached 93% and 92% at those respective intervals. Three participants received a second CALEC transplant, one of whom achieved complete success by the end of the study.
Read the full article here.
References
1. Jurkunas, U et al. “Cultivated Autologous Limbal Epithelial Cell (CALEC) Transplantation for Limbal Stem Cell Deficiency: a Phase I/II clinical trial of the first xenobiotic-free, serum-free, antibiotic-free manufacturing protocol developed in the US” Nature Communications DOI: 10.1038/s41467-025-56461-1