At the National Institutes of Health, a surgical team successfully implanted a patch of tissue created from patient cells with the goal of curing severe “dry” age-related macular degeneration (AMD), also known as geographic atrophy. Dry AMD is a prominent cause of vision loss among older Americans and currently has no cure.
The patient underwent the therapy as part of a clinical trial that is the first in the United States to use replacement tissues using patient-derived induced pluripotent stem (iPS) cells. The surgery was conducted by Amir H. Kashani, M.D., Ph.D., associate professor of ophthalmology, Wilmer Eye Institute, Johns Hopkins School of Medicine with support by Shilpa Kodati, M.D., staff clinician, NEI. The surgery was performed at the NIH Clinical Center in Bethesda, Maryland, under a phase 1/2a clinical trial to establish the therapy’s safety.
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. Safety and efficacy of this cell treatment was investigated by the NEI preclinical team. Clinical-grade production of this cell treatment was accomplished at the Center for Cellular Engineering, Department of Transfusion Medicine, Clinical Center, NIH.
This procedure represents the culmination of 10 years of research and development at the NEI. In the NIH lab, the patient’s blood cells were turned to iPS cells, which may become practically any type of cell in the body. In this case, they were engineered to become retinal pigment epithelial (RPE) cells, the type of cell that degenerates in the advanced types of dry AMD. RPE cells nourish and sustain light-sensing photoreceptors in the retina. In AMD, the loss of RPE leads to the loss of photoreceptors, which causes vision loss. This work was supported by the NIH Common Fund and NEI Intramural grants.
NIH Spokespeople:
Kapil Bharti, Ph.D., senior investigator, Ocular and Stem Cell Translational Research Section, NEI
Brian Brooks, M.D., Ph.D., chief, Ophthalmic Genetics and Visual Function Branch, NEI
To schedule interviews with Drs. Bharti and Brooks, email NEI at neinews@nei.nih.gov
More information:
NIH initiates first U.S. clinical trial of patient-derived stem cell therapy to replace and repair dying cells in retina (News release) (News release)
NIH researchers recover photoreceptors, avert blindness in animal models of retinal degeneration (News release) (News release)
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated with Age-Related Macular Degeneration (Clinical study details) (Clinical trial information)
About the NEI: NEI leads the federal government’s efforts to reduce vision loss and improve quality of life through vision research…
encouraging innovation, fostering cooperation, increasing the vision workforce, and educating the public and critical stakeholders. NEI supports basic and clinical science projects to discover sight-saving medicines and to enhance options for people with vision impairment. For more information, visit https://www.nei.nih.gov.
About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, contains 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the principal federal organization performing and supporting fundamental, clinical, and translational medical research, and is exploring the causes, treatments, and cures for both common and rare diseases. For more information on NIH and its initiatives, visit www.nih.gov.
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