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News Releases

Media Advisory

Wednesday, August 31, 2022

First U.S. patient receives autologous stem cell therapy to treat dry AMD

Image of RPE patch Left shows an image of the full-RPE-patch (2 x 4 mm). Each dot is an RPE cell with the borders stained green. Each patch contains approximately 75,000 RPE cells. Right image shows patch RPE cells at higher magnification.Kapil Bharti, Ph.D., NEI

What

At the 四虎影院, a surgical team successfully implanted a patch of tissue made from patient cells with the goal of treating advanced 鈥渄ry鈥 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.聽 The procedure was performed at the 四虎影院Clinical Center in Bethesda, Maryland, under a phase 1/2a clinical trial to determine the therapy鈥檚 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 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, NIH.

This surgery is the culmination of 10 years of research and development at the NEI. In the 四虎影院lab, the patient鈥檚 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 四虎影院Common Fund and NEI Intramural funding.

四虎影院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鈥檚 efforts to eliminate vision loss and improve quality of life through vision research鈥riving 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 四虎影院 (NIH): NIH, 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. 四虎影院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 四虎影院and its programs, visit www.nih.gov.

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