CALEC trial principal investigator Ula Jurkunas, MD, affiliate director of the Cornea Service at Mass Eye and Ear and professor of Ophthalmology at Harvard Medical Faculty, in her lab at Mass Eye and Ear in Boston. Credit score: Mass Eye and Ear
An expanded scientific trial that examined an experimental stem cell remedy for blinding cornea accidents discovered the remedy was possible and protected in 14 sufferers who had been handled and adopted for 18 months, and there was a excessive proportion of full or partial success.
The outcomes of this Part I/II trial are revealed in Nature Communications.
The remedy, known as cultivated autologous limbal epithelial cells (CALEC), was developed at Mass Eye and Ear. The revolutionary process consists of eradicating stem cells from a wholesome eye with a biopsy, increasing them right into a mobile tissue graft in a novel manufacturing course of that takes two to 3 weeks, after which surgically transplanting the graft into the attention with a broken cornea.
“Our first trial in four patients showed that CALEC was safe and the treatment was possible,” stated principal investigator Ula Jurkunas, MD, affiliate director of the Cornea Service at Mass Eye and Ear and professor of Ophthalmology at Harvard Medical Faculty.
“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.”
Researchers confirmed that CALEC utterly restored the cornea in 50% of individuals at their three-month go to, and that the speed of full success elevated to 79% and 77% at their 12- and 18-month visits, respectively.
With two individuals assembly the definition of partial success at 12 and 18 months, the general success of CALEC was 93% and 92% at 12 and 18 months. Three individuals obtained a second CALEC transplant, considered one of whom reached full success by the research finish go to. An extra evaluation of CALEC’s impression on imaginative and prescient confirmed various ranges of enchancment of visible acuity in all 14 CALEC sufferers.
CALEC displayed a excessive security profile, with no critical occasions occurring in both the donor or recipient eyes. One opposed occasion, a bacterial an infection, occurred in a single participant, eight months after the transplant resulting from power contact lens use. Different opposed occasions had been minor and resolved rapidly following the procedures.
CALEC stays an experimental process and is at the moment not supplied at Mass Eye and Ear or any U.S. hospital, and extra research can be wanted earlier than the remedy is submitted for federal approval.
Principal investigator Ula Jurkunas, MD, performs the primary stem cell transplantation of the CALEC scientific trial in 2018 at Mass Eye and Ear in Boston, on a affected person who suffered a extreme chemical burn in his eye. Credit score: Mass Eye and Ear
The CALEC trial is the primary human research of a stem cell remedy within the eye within the U.S. Different analysis collaborators embody Jia Yin, MD, Ph.D. and Reza Dana, MD at Mass Eye and Ear; Jerome Ritz, MD, of Dana-Farber Most cancers Institute’s Connell and O’Reilly Households Cell Manipulation Core Facility, the place the manufacturing of the stem cell graft takes place; and Myriam Armant, Ph.D., of Boston Youngsters’s Hospital; and the JAEB Middle for Well being Analysis.
The cornea is the clear, outermost layer of the attention. Its outer border, the limbus, accommodates a big quantity of wholesome stem cells known as limbal epithelial cells, which preserve the attention’s clean floor. When an individual suffers a cornea damage, similar to a chemical burn, an infection or different trauma, it will possibly deplete the limbal epithelial cells, which might by no means regenerate.
The ensuing limbal stem cell deficiency renders the attention with a completely broken floor the place it will possibly’t bear a corneal transplant, the present commonplace of look after imaginative and prescient rehabilitation. Folks with these accidents usually expertise persistent ache and visible difficulties.
This want led Jurkunas, a junior scientist, and Dana, director of the Cornea Service at Mass Eye and Ear, to discover a brand new method for regenerating limbal epithelial cells. Almost twenty years later, following preclinical research and collaborations with researchers at Dana-Farber and Boston Youngsters’s, it was potential to constantly manufacture CALEC grafts that met stringent high quality standards wanted for human transplantation.
The scientific trial was accepted by the U.S. Meals and Drug Administration (FDA) and Mass Normal Brigham Institutional Evaluation Board (IRB) and the primary affected person was handled in 2018 at Mass Eye and Ear. Profitable completion of the trial was achieved by way of shut coordination between Jurkunas’ surgical staff and the cell manufacturing facility at Dana-Farber.
Research like this present the promise of cell remedy for treating incurable situations. Mass Normal Brigham’s Gene and Cell Remedy Institute helps to translate scientific discoveries made by researchers into first-in-human scientific trials and, finally, life-changing therapies for sufferers.
As an autologous remedy, one limitation of this method is that it’s vital for the affected person to have just one concerned eye so a biopsy could be carried out to get beginning materials from the unaffected regular eye.
“Our future hope is to set up an allogeneic manufacturing process starting with limbal stem cells from a normal cadaveric donor eye,” stated Dr. Ritz “This will hopefully expand the use of this approach and make it possible to treat patients who have damage to both eyes.”
Within the interim, future CALEC research ought to embody bigger numbers of sufferers at a number of facilities, with longer follow-ups and a randomized-control design.
“We feel this research warrants additional trials that can help lead towards FDA approval,” stated 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.”
Extra info:
Jurkunas, U et al. Cultivated autologous limbal epithelial cell (CALEC) transplantation for limbal tem cell deficiency: a part I/II scientific trial of the primary xenobiotic-free, serum-free, antibiotic-free manufacturing protocol developed within the US, Nature Communications (2025). DOI: 10.1038/s41467-025-56461-1
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