Researchers from Mass Eye and Ear, a member of Mass General Brigham, have reported promising results from a phase I trial of a stem cell treatment for restoring vision in chemical burn patients. The treatment, called cultivated autologous limbal epithelial cell transplantation (CALEC), was found to be safe and well-tolerated in four patients with significant chemical burns in one eye.
According to a study published in Science Advances, the patients who were followed for 12 months experienced restored cornea surfaces. Two of them were able to undergo a corneal transplant, while the other two reported significant improvements in vision without further treatment.
The CALEC procedure involves removing stem cells from a patient’s healthy eye and growing them on a graft using an innovative manufacturing process. After two to three weeks, the graft is transplanted into the eye with corneal damage. This approach aims to address the limitations and risks associated with current treatment options for limbal stem cell deficiency, a condition caused by chemical burns and other eye injuries that results in permanent vision loss and discomfort.
The researchers consider the early findings of the phase I trial to be promising and are hopeful that CALEC can fill the treatment gap for patients with untreatable vision loss and pain associated with major cornea injuries. The CALEC project is a collaboration between researchers from Mass Eye and Ear, Dana-Farber Cancer Institute, Boston Children’s Hospital, and the JAEB Center for Health Research.
While autologous stem cell approaches have been described in previous studies, no U.S. research team had successfully developed a manufacturing process and quality control tests that met the requirements of the U.S. Food and Drug Administration (FDA) or demonstrated clinical benefits. The success of the CALEC procedure in the phase I trial is a significant breakthrough in this field.
The researchers are now preparing for the next phase of the clinical trial, which will involve tracking 15 CALEC patients for 18 months to further evaluate the efficacy of the procedure. Their goal is to establish CALEC as a viable treatment option for patients who were previously left with long-term deficits due to the severity of their eye injuries.
This study highlights the promise of cell therapy in treating previously incurable conditions. The Gene and Cell Therapy Institute at Mass General Brigham is at the forefront of translating scientific discoveries into life-changing treatments for patients. Its multidisciplinary approach enables researchers to advance new therapies and push the boundaries of this emerging field.
In conclusion, the phase I trial of the CALEC procedure for restoring vision in chemical burn patients has shown promising results. The early findings indicate that CALEC can offer hope to patients with untreatable vision loss and pain caused by major cornea injuries. Further research and clinical trials will be conducted to evaluate the procedure’s overall efficacy and its potential to fill the current treatment gap.
