Scientists Take Important Step Toward Using Retinal Cell Transplants to Treat Blindness
Latest discovery is promising step in use of cell therapy to treat retinal diseases
Retinal cells derived from a cadaver human eye survived when transplanted into the eyes of primate models, an important advance in the development of cell therapy to treat blindness, according to a study published on January 14 in Stem Cell Reports.
The retinal pigment epithelium (RPE), a layer of pigmented cells in the retina, functions as a barrier and regulator in the eye to maintain normal vision. RPE dysfunction can lead to eye disorders including macular degeneration and can cause blindness, which affects about 200 million people worldwide.
To restore this population of cells, the researchers extracted retinal stem cells from donated cadaver adult eyes, which can enable donor compatibility matching and can serve as a recurring source of human RPE. The team then assessed the safety and feasibility of implanting adult retinal stem cells into non-human primates.
The study found that RPE patches transplanted under the macula, or the central part of the retina, remained stable and integrated in vivo for at least three months without serious side effects such as immune attack or light sensitivity. The researchers also found that the stem cell-derived RPE at least partially took over the function of the original RPE and was able to support the endogenous photoreceptor, which helps with light and water absorption among other functions.
“We have demonstrated human cadaver donor-derived RPE at least partially replaces function in the macula of a non-human primate,” said the study’s co-lead investigator Timothy Blenkinsop, PhD, Assistant Professor of Cell, Developmental and Regenerative Biology and Ophthalmology, and member of the Black Family Stem Cell Institute and Eye and Vision Research Institute at the Icahn School of Medicine at Mount Sinai. “Human cadaver donor-derived cells can be safely transplanted underneath the retina and replace host function, and therefore may be a promising source for rescuing vision in patients with retina diseases. The results of this study suggest human adult donor RPE is safe to transplant, strengthening the argument for human clinical trials for treating retina disease.”
The researchers said transplantation of RPE stem cells derived from human adult cadaver eyes to replace the defective RPE is a possible treatment for macular degeneration, but additional research on this approach is necessary. Future studies should explore whether RPE stem cells derived from cadaver adult eyes can restore vision in human patients and diseased non-human primate models.
The study is an international collaboration between scientists from the Icahn School of Medicine at Mount Sinai, the Institute of Molecular and Cell Biology at the Agency of Science, Technology and Research in Singapore, the National University of Singapore, the Singapore Eye Research Institute, and the Knappschaft Hospital Saar in Germany.
About the Mount Sinai Health System
The Mount Sinai Health System is New York City's largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai advances medicine and health through unrivaled education and translational research and discovery to deliver care that is the safest, highest-quality, most accessible and equitable, and the best value of any health system in the nation. The Health System includes approximately 7,300 primary and specialty care physicians; 13 joint-venture ambulatory surgery centers; more than 415 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and more than 30 affiliated community health centers. The Mount Sinai Hospital is ranked on U.S. News & World Report's "Honor Roll" of the top 20 U.S. hospitals and is top in the nation by specialty: No. 1 in Geriatrics and top 20 in Cardiology/Heart Surgery, Diabetes/Endocrinology, Gastroenterology/GI Surgery, Neurology/Neurosurgery, Orthopedics, Pulmonology/Lung Surgery, Rehabilitation, and Urology. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 12 in Ophthalmology. Mount Sinai Kravis Children's Hospital is ranked in U.S. News & World Report’s “Best Children’s Hospitals” among the country’s best in four out of 10 pediatric specialties. The Icahn School of Medicine is one of three medical schools that have earned distinction by multiple indicators: ranked in the top 20 by U.S. News & World Report's "Best Medical Schools," aligned with a U.S. News & World Report "Honor Roll" Hospital, and No. 14 in the nation for National Institutes of Health funding. Newsweek’s “The World’s Best Smart Hospitals” ranks The Mount Sinai Hospital as No. 1 in New York and in the top five globally, and Mount Sinai Morningside in the top 20 globally.