US non-profit healthcare research system Mass General Brigham has announced that a Phase I/II trial of its stem cell therapy for cornea injuries has restored the cornea in 50% of patients after three months of treatment.
Published in Nature Communications, results saw the study achieve success rates of 79% and 77% corneal repair with the cultivated autologous limbal epithelial cells (CALEC) graft, at the 12-month and 18-month marks respectively. All enrolled patients who received the CALEC graft saw an improvement in visual acuity.
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The single-site, first-in-human Phase I/II trial (NCT02592330) enrolled 14 patients who received the graft. Three patients have since received a second stem cell graft, with one going on to see a completely successful cornea restoration.
CALECs are created by removing stem cells from a healthy eye with a biopsy, expanding them into a cellular tissue graft and surgically transplanting the graft into the eye. It does have limitations due to it being an autologous therapy, meaning that one eye must be healthy to provide viable stem cells for the graft. Mass Brigham hopes to develop an allogeneic therapy using cadaver donor eyes to make the approach accessible to patients with damage to both eyes.
Principal investigator Ula Jurkunas said: “Our first trial in four patients showed that CALEC was safe and the treatment was possible. 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 trial is the first to be jointly funded by the National Institutes of Health’s (NIH) National Eye Institute (NEI). According to Mass General Brigham, CALEC is the first eye-based stem cell therapy in the US.
Researchers developed CALEC by taking cells from the limbus section of the still functioning cornea, meaning the therapy would require the patient to have one fully functioning eye. Researchers took limbal epithelial cells, which maintain the eye’s smooth surface and are unable to regenerate, using them to create a graft that can be applied to the affected eye. The non-profit health group selected the Dana-Farber Cancer Institute to manufacture the surgical grafts.
Jurkunas added: “We feel this research warrants additional trials that can help lead towards US Food and Drug Administration (FDA) approval. While we are proud to have been able to bring a new treatment from the lab bench to clinical trials, our guiding objective was and always will be for patients around the country to have access to this effective treatment.”
Elsewhere in gene-based eye care, MeiraGTx is advancing its investigational gene therapy toward expedited approval in the UK after 11 children who were born blind from birth gained significant vision improvements. Meanwhile, Emmecell has published positive topline outcomes from a randomised Phase I extension trial of EO2002, its non-surgical cell therapy candidate for treating corneal oedema.
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