A groundbreaking study from Mass Eye and Ear reveals promising results in the use of stem cell therapy for treating severe corneal injuries, offering hope for patients with otherwise untreatable conditions. The Phase I/II clinical trial, which involved 14 participants, showed that transplants of stem cells from a patient’s healthy eye successfully restored damaged corneal surfaces, with many participants experiencing significant improvements in their vision.
The cornea, the transparent outer layer of the eye, plays a crucial role in protecting the eye and focusing light onto the retina. Serious injuries or infections can scar the cornea, and while corneal grafts—transplants of healthy tissue from donors—are commonly used to treat these conditions, some injuries are too extensive for this method to be effective. In cases of limbal stem cell deficiency, the cornea’s supply of replenishing cells is depleted, leading to irreversible damage that standard grafts cannot address.
Lead researcher Dr. Ula Jurkunas, an associate director at the Cornea Service at Mass Eye and Ear, explained that limbal stem cell deficiency often results in significant vision loss, intense pain, and a lack of viable treatment options. “When people have a corneal stem cell deficiency, they can have a really white cornea and no vision. There are really no good ways to treat that,” she said.
To address this, the research team developed a novel technique to harvest and grow healthy stem cells from an unaffected part of the patient’s cornea. These cultivated autologous limbal epithelial cells (CALEC) are then used to create a cellular graft, which is transplanted onto the injured cornea. Previous studies by Jurkunas and her team indicated that CALEC grafts were both safe and effective in the short term.
The recent findings, published in Nature Communications, follow 14 patients who were monitored for up to 18 months post-procedure. Results were promising, with 92% of participants showing at least a partial improvement in their corneal health. In 77% of cases, there was a complete restoration of the corneal surface, with some patients experiencing enhanced vision. Although one patient did encounter a bacterial infection months after the procedure, it was attributed to chronic contact lens use, rather than the transplant itself.
“This procedure has had transformative effects for many patients,” said Jurkunas. “They had severe injuries that were previously untreatable. One patient even told me, ‘I actually got my life back.'”
While still considered experimental, this therapy represents the first stem cell treatment in the U.S. to successfully restore corneal vision in patients suffering from corneal blindness. The procedure uses stem cells derived from the patient’s own body, a significant step forward compared to other therapies that rely on embryonic stem cells.
The researchers are now focused on expanding their clinical trials and refining the technique to possibly include stem cells from other donors. This advancement could help patients with damage to both corneas, potentially making the therapy available to a broader range of individuals. If ongoing trials continue to demonstrate success, CALEC could become a standard treatment for cases of corneal blindness that were once deemed irreversible.
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