Stem Cell Therapy May Help Restore Vision in Patients With Eye Injuries

An experimental stem cell procedure appears to safely treat vision loss in patients with eye injuries resulting from chemical burns, according to findings from a small Phase 1 study published last week in Science Advances. A team of researchers led by Mass Eye and Ear tested the procedure, called cultivated autologous limbal epithelial cell transplantation (CALEC), in four patients who developed limbal stem cell (LSC) deficiency in one eye due to significant chemical burns.

LSC deficiency is an irreversible loss of cells on the tissue surrounding the cornea that can cause permanent vision loss, pain and discomfort in the affected eye. Furthermore, without limbal cells and a healthy eye surface, these patients are unable to undergo artificial cornea transplants, the current standard of vision rehabilitation.

With CALEC, stem cells from a patient’s healthy eye are removed via a small biopsy and then expanded and grown on a graft. After two to three weeks, the graft is transplanted into the eye with corneal damage. In the study. patients were followed for 12 months. Each of the four patients saw early positive results after the procedure. Two were able to undergo a corneal transplant and two reported significant improvements in vision without additional treatment.

“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 Ula Jurkunas, MD, principal investigator and lead study author. “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.”

Jurkunas and her co-investigators are now finalizing the next phase of the clinical trial: tracking 15 CALEC patients for 18 months to better determine the procedure’s overall efficacy. Their hope is that CALEC can one day become a treatment option for patients who previously had to endure long-term deficits when existing treatments were not an option.