Trials to begin for stem cell-based therapy to treat geographic atrophy

A National Eye Institute (NEI) study will test the safety of a stem cell treatment for the dry (atrophic) form of age-related macular degeneration (AMD).

The researchers will take a patient’s own blood cells, and in a lab, convert them into induced pluripotent stem cells (iPSC) capable of becoming any type of cell in the body. The iPS cells are then programmed to become retinal pigment epithelial (RPE) cells, which nourish the photoreceptor sight cells.

Before they are transplanted, the iPSC-derived RPE are grown in sheets one cell thick, replicating their natural structure within the eye. This monolayer of iPSC-derived RPE is grown on a biodegradable scaffold designed to promote the integration of the cells within the retina. Surgeons position the patch between the RPE and the photoreceptors using a surgical tool designed specifically for that purpose.

Under the phase I/IIa clinical trial protocol 12 patients with advanced-stage geographic atrophy will receive the iPSC-derived RPE implant in one of their eyes and be closely monitored for a period of at least one year to confirm safety before moving into later phases.

This is the first clinical trial in the U.S. to use replacement tissues from patient-derived iPS cells. To keep up on latest research for dry (atrophic) AMD, see A Guide to Research in Dry AMD on this site.