Patient's own stem cells heal cornea damage using new method

CALEC procedure restored the cornea surfaces of four patients with severe chemical burns safely in the Phase I trial
Shubhangi Dua
New procedure shows promise in healing damage cornea surface
New procedure shows promise in healing damage cornea surface

Zarina Lukash / iStock 

Scientists developed the cultivated autologous limbal epithelial cells (CALEC) procedure to treat unilateral limbal stem cell (LSC) deficiency.

Past research explains that LSC deficiency often leads to cloudy corneas caused by stem cell damage leaving impaired vision. Furthermore, injuries or diseases damage these cells, causing additional vision problems.

A team of scientists from Mass Eye and Ear, a member of Mass General Brigham, are addressing this medical condition and found a possibly effective solution to repair cornea damage.

CALEC procedure findings

They developed a technique called – cultivated autologous limbal epithelial cell transplantation (CALEC) that used the patient's own stem cells from the healthy eye and successfully repaired the cornea surface in the affected eye. 

The treatment was found to be safe and feasible in the study’s Phase I trial when tested on four patients with significant chemical burns in one eye.

A statement by the researchers reported that the patients who were followed for 12 months experienced restored cornea surfaces – two were able to undergo a corneal transplant, and two reported significant improvements in vision without additional treatment.

Ula Jurkunas, MD, associate director of the Cornea Service at Mass Eye and Ear and an associate professor of ophthalmology at Harvard Medical School, stated: 

“Our early results suggest that CALEC might offer hope to patients who had been left with untreatable vision loss and pain associated with major cornea injuries. Cornea specialists have been hindered by a lack of treatment options with a high safety profile to help our patients with chemical burns and injuries that render them unable to get an artificial cornea transplant. We are hopeful with further study, CALEC can one day fill this crucially needed treatment gap.”

Healthy eye stem cell restores sight

The procedure required stem cells from a patient's healthy eye to be removed by performing a small biopsy which was then expanded and grown on a graft, according to the statement.

The CALEC procedure graft was sent back after two to three weeks to the medical center, and it was transplanted into the eye with corneal damage. 

Addressing the challenges of innovating the technique, 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, stated:

“It was challenging to develop a process for creating limbal stem cell grafts that would meet the FDA’s strict regulatory requirements for tissue engineering. Having developed and implemented this process, it was very gratifying to see encouraging clinical outcomes in the first cohort of patients enrolled in this clinical trial.”

The statement announced that the researchers are preparing for the next trial phase, observing 15 CALEC patients for 18 months and determining the treatment’s overall efficacy. 

Researchers envision the CALEC procedure to become a treatment option for patients who previously had to endure long-term deficits when existing treatment options were not an option given the severity of their injuries.

“We are inspired by a desire to provide patients who have limited treatment options with better possibilities for care, and Dr. Jurkunas and her colleagues at Mass Eye and Ear and across multiple academic centers have dedicated nearly two decades' worth of work towards one day achieving this goal for people with significant corneal injuries,” said Joan W Miller, chair of Ophthalmology at Mass Eye and Ear and Mass General Hospital and Professor of Ophthalmology at Harvard Medical School. 

The study was published on August 18 in the journal Science Advances

Study abstract:

To treat unilateral limbal stem cell (LSC) deficiency, we developed cultivated autologous limbal epithelial cells (CALEC) using an innovative xenobiotic-free, serum-free, antibiotic-free, two-step manufacturing process for LSC isolation and expansion onto human amniotic membrane with rigorous quality control in a good manufacturing practices facility. Limbal biopsies were used to generate CALEC constructs, and final grafts were evaluated by noninvasive scanning microscopy and tested for viability and sterility. Cultivated cells maintained epithelial cell phenotype with colony-forming and proliferative capacities. Analysis of LSC biomarkers showed preservation of “stemness.” After preclinical development, a phase 1 clinical trial enrolled five patients with unilateral LSC deficiency. Four of these patients received CALEC transplants, establishing preliminary feasibility. Clinical case histories are reported, with no primary safety events. On the basis of these results, a second recruitment phase of the trial was opened to provide longer term safety and efficacy data on more patients.

Add Interesting Engineering to your Google News feed.
Add Interesting Engineering to your Google News feed.
message circleSHOW COMMENT (1)chevron
Job Board