NIH to Launch First U.S. Clinical Trial Patient-Derived Stem Cell Therapy to Replace Dying Cells in Retina

NIH to Launch First U.S. Clinical Trial Patient-Derived Stem Cell Therapy to Replace Dying Cells in Retina

Researchers at the National Eye Institute (NEI) will launch a clinical trial to test the safety of a novel patient-specific stem cell-based therapy to treat geographic atrophy, the advanced “dry” form of age-related macular degeneration (AMD), a leading cause of vision loss among people age 65 and older. There is no treatment for the geographic atrophy form of AMD.

A protocol which prevented blindness in preclinical research on animals, this will be the first clinical trial in the United States to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC), reports Kapil Bharti, PhD, a senior investigator and head of the NEI Ocular and Stem Cell Translation Research Section. The NEI is part of the National Institutes of Health (NIH).

The Regenerative Therapy

This regenerative medicine-based therapy involves taking a patient’s blood cells and, in a lab, converting them into iPS cells, which have the potential to form any type of cell in the body. The iPS cells are programmed to become retinal pigment epithelial (RPE) cells, the type of cell that dies early in the geographic atrophy stage of macular degeneration. RPE cells nurture photoreceptors, the ...

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