Scientists have shared promising findings from a phase I clinical trial centred on an advanced stem cell therapy for repairing cornea damage.
The innovative therapy, known as cultivated autologous limbal epithelial cell transplantation (CALEC), demonstrates both safety and short-term efficacy in patients suffering from severe cornea damage caused by chemical burns.
Published in the journal Science Advances, the team’s study showcases the potential of CALEC in transforming the lives of patients previously deemed untreatable due to their extensive cornea injuries.
The trial tracked four patients over 12 months and revealed remarkable outcomes.
Two individuals could undergo successful corneal transplants, while the remaining two reported significant enhancements in vision without requiring supplementary interventions.
Though the primary aim of the phase I trial was to assess the therapy's initial safety and feasibility, the researchers are optimistic about its implications.
Principal investigator Dr Ula Jurkunas, who is also the associate director of the Cornea Service at Mass Eye and Ear and an associate professor of ophthalmology at Harvard Medical School, emphasised the potential of CALEC to address the critical treatment gap for patients enduring vision loss and pain due to major cornea injuries.
The CALEC procedure involves harvesting stem cells from a patient’s healthy eye through a minimally invasive biopsy.
These cells are then expanded and cultured on a graft using an innovative manufacturing process at the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute.
After two to three weeks, the cultivated graft is transplanted back into the damaged eye at Mass Eye and Ear.
This ground-breaking collaboration is the brainchild of Dr Jurkunas and her peers at the Cornea Service at Mass Eye and Ear, alongside researchers from Dana-Farber Cancer Institute, Boston Children’s Hospital, and the JAEB Centre for Health Research.
The clinical trial, noteworthy for being the first human study of a stem cell therapy funded by the National Eye Institute (NEI), signifies a pivotal step in advancing treatment options for patients suffering from chemical burns and related eye injuries.
Chemical burns and similar ocular traumas can result in limbal stem cell deficiency, a condition marked by the irreversible loss of cells around the cornea.
This often leads to chronic vision impairment, pain, and discomfort in the affected eye. The CALEC procedure looks to revolutionise treatment by utilising a patient's stem cells, which are then cultured and expanded to create a cell sheet that facilitates tissue growth.
Dr Jerome Ritz, executive director of the Connell and O'Reilly Families Cell Manipulation Core Facility at Dana-Farber and professor of medicine at Harvard Medical School, highlighted the challenges in creating a manufacturing process that meets stringent FDA regulations for tissue engineering.
The successful implementation of this process and the promising clinical outcomes witnessed in the trial’s initial cohort of patients mark a significant achievement.
As studies like these continue to underscore the potential of cell therapies in tackling previously incurable conditions, the Gene and Cell Therapy Institute at Mass General Brigham plays a pivotal role in translating scientific breakthroughs into tangible clinical trials.
This multidisciplinary approach positions the institute at the forefront of pioneering treatments, pushing the boundaries of technology and clinical application.
The phase I trial enrolled five patients with chemical burns to one eye, with four receiving the CALEC treatment. Detailed quality control assessments determined that the fifth patient's cells were unsuitable for expansion.
Over a 12-month follow-up period, the CALEC recipients demonstrated remarkable progress. One patient regained vision sufficiently for an artificial cornea transplant, while another experienced a substantial improvement from 20/40 to 20/30 vision.
The team is preparing for the next phase of clinical trials involving 15 CALEC patients, with an 18-month observation period to evaluate the therapy's overall efficacy.
There are high hopes that CALEC could eventually fill a crucial void in eye injury treatments, offering a lifeline to patients who have endured long-term deficits due to the lack of viable options.
Picture caption - Ula Jurkunas (left) at a monitor with researchers in her laboratory
