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Cell therapy that repairs cornea damage with patient’s own stem cells achieves positive Phase I trial results

Cultivated autologous limbal epithelial cells (CALEC) procedure shown to be safe and feasible with early positive results of restored cornea surfaces or vision gains in four patients with severe chemical burns
August 18, 2023
Physician performing surgery in an operating room.

Ula Jurkunas, M.D., performs the first CALEC transplant in the United States as part of a clinical trial at Mass Eye and Ear in April 2018. Image credit: Mass Eye and Ear

A team led by researchers from Mass Eye and Ear, a member of Mass General Brigham, reports the results of a phase I trial of a revolutionary stem cell treatment called cultivated autologous limbal epithelial cell transplantation (CALEC), which was found to be safe and well-tolerated over the short term in four patients with significant chemical burns in one eye. According to the study published August 18 in Science Advancesthe patients who were followed for 12 months experienced restored cornea surfaces — two were able to undergo a corneal transplant and two reported significant improvements in vision without additional treatment.

While the phase I study was designed to determine preliminary safety and feasibility before advancing to a second phase of the trial, the researchers consider the early findings promising.

“Our early results suggest that CALEC might offer hope to patients who had been left with untreatable vision loss and pain associated with major cornea injuries,” said principal investigator and lead study author Ula Jurkunas, MD, associate director of the Cornea Service at Mass Eye and Ear and an associate professor of ophthalmology at Harvard Medical School. “Cornea specialists have been hindered by a lack of treatment options with a high safety profile to help our patients with chemical burns and injuries that render them unable to get an artificial cornea transplant. We are hopeful with further study, CALEC can one day fill this crucially needed treatment gap.”