First U.S. Patient Receives Autologous Stem Cell Therapy to Treat Macular Degeneration

A surgical team at the National Institutes of Health (NIH) successfully implanted a patch of tissue made from autologous induced pluripotent stem cells (iPSC) to treat advanced “dry” age-related macular degeneration, the agency announced last week. The surgery, performed as part of a phase 1/2a clinical trial, is the first in the United States to use replacement tissues from autologous iPSCs.

According to NIH, the surgery represents the culmination of 10 years of research and development at the National Eye Institute, part of the NIH. In the NIH lab, the patient’s blood cells were converted to iPSCs and then programmed to become retinal pigment epithelial (RPE) cells, the type of cell that degenerates in the advanced forms of dry AMD.

This iPSC-derived therapy was developed by the Ocular and Stem Cell Translational Research Section team led by Kapil Bharti, PhD, senior investigator at the NEI, in collaboration with FUJIFILM Cellular Dynamics Inc., and Opsis Therapeutics. Safety and efficacy of this cell therapy were 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.

Additional information about the trial is available online.