Eye On the Cure Research News

Dec 17, 2019

First Human Study in US for Induced Pluripotent Stem Cells to be Launched for Advanced Dry AMD

Currently there are no treatments for Advanced Dry AMD, also known as GA, which can lead to significant central vision loss

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The National Eye Institute (NEI), one of the federally funded National Institutes of Health, is launching the first clinical trial in the US for induced pluripotent stem cells (iPSC), stem cells that are derived from mature human cells. The iPSC in the landmark study are being derived from the patient’s own blood and developed into retinal pigment epithelial (RPE) cells for the treatment of geographic atrophy (GA), the advanced form of dry age-related macular degeneration (AMD), which causes progressive central vision loss.

The 12-person, Phase 1/2a, safety trial will be conducted at the NEI in Bethesda, Maryland.

The hallmark of GA is the loss of RPE cells, a single layer of cells which provide essential nutrients and waste disposal for photoreceptors, the cells that make vision possible. When RPE cells degenerate or don’t function properly, photoreceptors in the macula, the center of the retina, are lost.

By replacing a patient’s lost or dysfunctional RPE cells, researchers hope to save and possibly restore their photoreceptors, and therefore, their vision.

By using a patient’s own iPSC to produce RPE cells — rather than RPE cells derived from embryonic stem cells — the researchers believe they will minimize the chance of cell rejection.

The sheet of RPE cells for the emerging treatment are grown on a biodegradable scaffold to promote their integration when surgically placed between the patient’s own RPE cells and photoreceptors.

To learn more about the clinical trial and treatment approach, see the video below featuring Kapil Bharti, a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section.