National Institute of Health launches first U.S. clinical trial of patient-derived stem cell therapy to replace dying cells in retina

Researchers at the National Eye Institute (NEI) are launching 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. The geographic atrophy form of AMD currently has no treatment.

“The protocol, which prevented blindness in animal models, is the first clinical trial in the U.S. to use replacement tissues from patient-derived induced pluripotent stem cells (iPSC),” said Kapil Bharti, Ph.D., a senior investigator and head of the NEI  Ocular and Stem Cell Translational Research Section. The NEI is part of the National Institutes of Health.