1Siriraj Center for Regenerative Medicine, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.Ratchapong Netsrithong 1,2 Methichit Wattanapanitch 1* NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Ībout the National Institutes of Health (NIH): NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. NIH researchers rescue photoreceptors, prevent blindness in animal models of retinal degeneration (News release)Īutologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated with Age-Related Macular Degeneration (Clinical trial information)Ībout the NEI: NEI leads the federal government’s efforts to eliminate vision loss and improve quality of life through vision research…driving innovation, fostering collaboration, expanding the vision workforce, and educating the public and key stakeholders. clinical trial of patient-derived stem cell therapy to replace and repair dying cells in retina (News release) Bharti and Brooks, contact NEI at More information Kapil Bharti, Ph.D., senior investigator, Ocular and Stem Cell Translational Research Section, NEIīrian Brooks, M.D., Ph.D., chief, Ophthalmic Genetics and Visual Function Branch, NEI This work was supported by the NIH Common Fund and NEI Intramural funding. In AMD, the loss of RPE leads to the loss of photoreceptors, which causes vision loss. RPE cells nourish and support light-sensing photoreceptors in the retina. In this case, they were programmed to become retinal pigment epithelial (RPE) cells, the type of cell that degenerates in the advanced forms of dry AMD. In the NIH lab, the patient’s blood cells were converted to iPS cells, which can become almost any type of cell in the body. This surgery is the culmination of 10 years of research and development at the NEI. Clinical-grade manufacturing of this cell therapy was performed at the Center for Cellular Engineering, Department of Transfusion Medicine, Clinical Center, NIH. Safety and efficacy of this cell therapy was tested by the NEI preclinical team. This iPS cell derived therapy was developed by the Ocular and Stem Cell Translational Research Section team led by Kapil Bharti, Ph.D., senior investigator at the National Eye Institute (NEI), part of NIH, in collaboration with FUJIFILM Cellular Dynamics Inc., and Opsis Therapeutics, based in Madison, Wisconsin. The procedure was performed at the NIH Clinical Center in Bethesda, Maryland, under a phase 1/2a clinical trial to determine the therapy’s safety. Kashani, M.D., Ph.D., associate professor of ophthalmology, Wilmer Eye Institute, Johns Hopkins School of Medicine with assistance by Shilpa Kodati, M.D., staff clinician, NEI. The patient received the therapy as part of a clinical trial that is the first in the United States to use replacement tissues from patient-derived induced pluripotent stem (iPS) cells. Dry AMD is a leading cause of vision loss among older Americans and currently has no treatment. Kapil Bharti, Ph.D., NEI WhatĪt the National Institutes of Health, a surgical team successfully implanted a patch of tissue made from patient cells with the goal of treating advanced “dry” age-related macular degeneration (AMD), also known as geographic atrophy. Right image shows patch RPE cells at higher magnification. Each patch contains approximately 75,000 RPE cells. Each dot is an RPE cell with the borders stained green. Left shows an image of the full-RPE-patch (2 x 4 mm).
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