46 EyeWorld Asia-Pacific | March 2025 CORNEA Dr. Koo said she’s excited to see progress in this field by other companies as well, noting the progress of Aurion Biotech’s therapy, which shows that cell therapy will be a game changer. She said that there have been developments beyond magnetic cell therapy for corneal disease, noting that magnetic cell delivery for macular degeneration is in development and stands to restore vision for another large group of patients suffering from vision loss. Cultivated Autologous Limbal Epithelial Cells (CALEC) Therapy Ula Jurkunas, MD, said there are a lot of cell therapy options looking at the corneal endothelium, the inner layer of the cornea. “The cell therapy that we have developed is for corneal epithelium, or the surface cells,” she said. “Corneal epithelial cells get destroyed due to chemical injuries, various infections, genetic disorders, even contact lens wear.” This can destroy the inside, or peripheral corneal epithelial cells, where the corneal stem cells reside. When there is corneal stem cell destruction, the patient develops limbal stem cell deficiency, the cornea becomes opaque, the patient can become blind, and it can be very painful, Dr. Jurkunas said. “We’ve developed technology where we are harvesting a small biopsy from healthy eyes. Let’s say it’s asymmetric disease—one eye is affected and the other is mostly healthy; we take them to a special GMP lab, and we isolate those stem cells, expand them, and make a lot more of them on a sheet.” CALEC involves cells on a sheet of amniotic membrane as a scaffold, then they are transplanted to the eye that has the injury or infection. Dr. Jurkunas noted that the first feasibility and safety study was published in Science Advances,1 and the efficacy paper study of the Phase 2 trial has been accepted in Nature Communications. The study showed that 92% of CALEC grafts achieved partial or complete success at 18 months of follow-up. The trial was the first of this kind in the U.S., though cultivated epithelial stem cells have been used outside the U.S. in the past. “We were the first center to develop this trial, and we did the trial without the support of industry,” she said. She added that it was the first trial of this kind using epithelial cells that are autologous, so there isn’t any rejection. Dr. Jurkunas said that the next step will be to go to the FDA to determine if a Phase 3 clinical trial is needed. Most therapies require a Phase 3 trial, she said, adding that the therapy has so far shown that it’s safe and feasible. One key factor still to be determined is where manufacturing for this therapy would occur—at one center or multiple centers in the country. It does require quite a lot of resources to manufacture it, she said. Dr. Jurkunas noted that this therapy is different from a standard corneal transplant. With the disease, a standard corneal transplant is going to fail right away because you don’t have healthy stem cells, so patients in need of this therapy may need a regular transplant and this transplant together. Looking to future developments with this treatment, Dr. Jurkunas said a next step could be investigating how to do this with allogeneic cells, for patients who have bilateral LSCD. A treatment like this would take cells from a cadaveric donor and culture, possibly with similar methods or with modifications. This would be helpful, she said, because of the prevalence of bilateral cases, but by using another donor, you would need to factor in the possibility of rejection as well. This is a stepping stone for cell therapy in the U.S., she said. “Our regulatory requirements are stringent, and this is the first time we’re using stem cell therapy in cornea.” She’s interested to see the other therapy options being developed for endothelial cells as well. If you have a cell therapy for endothelial cells, you wouldn’t need as many corneal cells or cadaveric donors. “There are regulatory hurdles that one has to overcome, but once we have one product, it’s always easier for other products to get approved and other technologies to be developed, so I think it’s definitely a stepping stone for that,” she said.
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