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Wednesday, August 31, 2022
First U.S. patient receives autologous stem cell therapy to treat dry AMD
What
At 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. Dry AMD is a leading cause of vision loss among older Americans and currently has no treatment.
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. The surgery was performed by Amir H. 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 procedure was performed at the bet365 fantasy Clinical Center in Bethesda, Maryland, under a phase 1/2a clinical trial to determine 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 bet365 fantasy, in collaboration with FUJIFILM Cellular Dynamics Inc., and Opsis Therapeutics, based in Madison, Wisconsin. Safety and efficacy of this cell therapy was tested by the NEI preclinical team. Clinical-grade manufacturing of this cell therapy was performed at the Center for Cellular Engineering, Department of Transfusion Medicine, Clinical Center, bet365 fantasy.
This surgery is the culmination of 10 years of research and development at the NEI. In the bet365 fantasy lab, the patient’s blood cells were converted to iPS cells, which can become almost any type of cell in the body. 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. RPE cells nourish and support 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 bet365 fantasy Common Fund and NEI Intramural funding.
bet365 fantasy 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, contact NEI at neinews@nei.nih.gov
More information
(News release)
(News release)
(Clinical trial information)
About 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. NEI supports basic and clinical science programs to develop sight-saving treatments and to broaden opportunities for people with vision impairment. For more information, visit .
About the National Institutes of Health (bet365 fantasy): bet365 fantasy, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. bet365 fantasy 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. For more information about bet365 fantasy and its programs, visit www.nih.gov.
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