Researchers find stem cells in normal and Fuchs corneal endothelium

October 03, 2016

Boston, Mass. -- Researchers from Massachusetts Eye and Ear have, for the first time, identified rapidly proliferating cells (known as "neural crest-derived progenitor cells") in the corneal endothelium of specimens from normal corneas and from corneas with Fuchs' Endothelial Corneal Dystrophy (FECD), a condition in which the cells responsible for keeping the cornea clear die prematurely -- often leading to blindness. The findings, published online today in the American Journal of Pathology, hold promise for new therapies to be developed using the proliferating cells to return normal clearing abilities to the cornea in patients with FECD.

"Previously, we thought that all of the cells in the corneal endothelium were unable to divide, but we were surprised to find this small population of dormant stem cells capable of proliferating," said principal investigator Ula Jurkunas, M.D. (in photo), a corneal and refractive surgeon at Massachusetts Eye and Ear and Associate Professor of Ophthalmology at Harvard Medical School. "We showed with various markers that they are progenitor cells. In addition, we were able to differentiate these cells into neurons-- and that's a characteristic of all neural crest-derived tissue and its progenitors"

The corneal endothelium is responsible for pumping water throughout the cornea to keep vision clear, and when cells of the corneal endothelium die, they do not regenerate. Accelerated cell loss in patients with FECD prevents clearing of the cornea, and if left untreated, may progress to blindness. An age-related and genetic eye disease, FECD is one of the most common causes of corneal swelling. It affects up to four percent of the American population over the age of 40, and it is becoming more prevalent in women.

Full or partial corneal transplants are the most often recommended course of treatment to restore lost vision in patients with FECD. However, a few clinical studies have shown that, by stripping away a central portion of the diseased cells, the surrounding cells can grow in to clear the cornea. The proliferating cells described in the American Journal of Pathology paper may explain why this method, known as Descemet-stripping, has been successful in some patients.

Additionally, the newly described proliferating cells hold the promise of being able to regenerate a person's own endothelial cells, even in cases of FECD, as an alternative to transplanting corneas from a donor. The findings may change the clinical approach to patients with FECD, who are currently being treated as being irreversibly damaged, requiring transplants to provide a new source of healthy cells.

"Our research shows that some cells in Fuchs' dystrophy are still healthy, and that means that we could potentially stimulate them to divide and regenerate the corneas in a diseased state," said Dr. Jurkunas.
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Copies of this paper are available to credentialed journalists upon request; please contact Elsevier's Newsroom at newsroom@elsevier.com or +31 20 485 2492.

Authors on the American Journal of Pathology paper include Dr. Jurkunas, Kishore Reddy Katikireddy, Ph.D., and Thore Schmedt, Ph.D., of Schepens Eye Research Institute of Massachusetts Eye and Ear, Marianne O. Price Ph.D., of the Cornea Research Foundation of America and Francis W. Price, M.D., of Price Vision Group. Research supported by Falk Medical Research Foundation, Cornea Donor Research Fund and R01EY020581.

About Massachusetts Eye and Ear

Mass. Eye and Ear clinicians and scientists are driven by a mission to find cures for blindness, deafness and diseases of the head and neck. Now united with Schepens Eye Research Institute, Mass. Eye and Ear is the world's largest vision and hearing research center, developing new treatments and cures through discovery and innovation. Mass. Eye and Ear is a Harvard Medical School teaching hospital and trains future medical leaders in ophthalmology and otolaryngology, through residency as well as clinical and research fellowships. Internationally acclaimed since its founding in 1824, Mass. Eye and Ear employs full-time, board-certified physicians who offer high-quality and affordable specialty care that ranges from the routine to the very complex. In the 2015-2016 "Best Hospitals Survey," U.S. News & World Report ranked Mass. Eye and Ear #1 in the nation for ear, nose and throat care and #1 in the Northeast for eye care. For more information about life-changing care and research, or to learn how you can help, please visit MassEyeAndEar.org.

About Harvard Medical School Department of Ophthalmology

The Harvard Medical School (HMS) Department of Ophthalmology is one of the leading and largest academic departments of ophthalmology in the nation. More than 350 full-time faculty and trainees work at ten HMS affiliate institutions, including Massachusetts Eye and Ear, Massachusetts General Hospital, Brigham and Women's Hospital, Boston Children's Hospital, Beth Israel Deaconess Medical Center, Joslin Diabetes Center/Beetham Eye Institute, Veterans Affairs Boston Healthcare System, VA Maine Healthcare System, and Cambridge Health Alliance. Formally established in 1871, the department has been built upon a strong and rich foundation in medical education, research, and clinical care. Through the years, faculty and alumni have profoundly influenced ophthalmic science, medicine, and literature--helping to transform the field of ophthalmology from a branch of surgery into an independent medical specialty at the forefront of science.

About American Journal of Pathology

The American Journal of Pathology, official journal of the American Society for Investigative Pathology seeks to publish high-quality, original papers on the cellular and molecular biology of disease. The editors accept manuscripts that advance basic and translational knowledge of the pathogenesis, classification, diagnosis, and mechanisms of disease, without preference for a specific analytic method. High priority is given to studies on human disease and relevant experimental models using cellular, molecular, animal, biological, chemical, and immunological approaches in conjunction with morphology.

Massachusetts Eye and Ear Infirmary

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