A groundbreaking experimental stem cell treatment for blinding corneal injury was found to be feasible and safe in patients who were followed for 18 months, with a high proportion of complete or partial success (over 90%) at restoring corneal surface and improving vision. The results of the phase ½ clinical trial have been published in Nature Communications, inspiring hope and warrant additional trials.
The CALEC Procedure
This procedure was developed to help with corneal injuries like chemical burns, infections, or other trauma that can lead to irreversible limbal stem cell deficiency. The novel procedure is called the cultivated autologous limbal epithelial cells, or CALEC for short. It involves taking stem cells from a healthy eye, which are expanded onto a graft over several weeks before being transplanted into a patient’s damaged eye.
“Our first trial in four patients showed that CALEC was safe and the treatment was possible,” said principal investigator Ula Jurkunas, MD, associate director of the Cornea Service at Mass Eye and Ear and professor of Ophthalmology at Harvard Medical School. “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.”
Repairing Coneal Damage
The results from the CALEC trial showed the treatment completely restored corneal damage in 50% of the participants at the 3-month check-up visit, and the rate of complete success increased to 79% at the one-year check-up, and to 77% at the 18-month check-up.
Two of the participants met the definition of partial success at 12 and 18 months, and the overall success of CALEC was 93% at 12 months and 92% at 18 months. Three participants received a second corneal CALEC transplant, of which one experienced complete success by the end check-up visit of the study.
Additional analysis of the impact of CALEC on vision showed varying levels of improvement of visual acuity in all 14 of the participants. The corneal procedure displayed a high safety profile with no adverse events occurring. However, one participant had a bacterial infection eight months after transplant due to chronic contact lens use. Any other adverse events were minor and were resolved quickly.
The CALEC trial is the first human study of a stem cell therapy to be funded by the National Eye Institute (NEI) branch of the NIH. However, the CALEC procedure remains an experimental procedure and it is not offered at Mass Eye and Ear or at any other hospital in America. Mass General Brigham’s Gene and Cell Therapy Institute will be conducting additional randomized-control design studies including a larger number of participants at multiple centers, with longer follow-ups before this treatment will be submitted for federal approval.
“Our future hope is to set up an allogeneic manufacturing process starting with limbal stem cells from a normal cadaveric donor eye,” said Jerome Ritz, MD, of Dana-Farber Cancer Institute’s Connell and O’Reilly Families Cell Manipulation Core Facility, where the manufacturing of the stem cell graft takes place. “This will hopefully expand the use of this approach and make it possible to treat patients who have damage to both eyes.”
“We feel this research warrants additional trials that can help lead towards FDA approval,” said Jurkunas. “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.”
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