Share In December of 2010, the FDA approved the first clinical trial using embryonic stem cells to treat macular degeneration.

This month, the first patients were treated at UCLA's Jules Stein Eye Institute. This clinical trial will enroll 24 patients - 12 with dry age-related macular degeneration and 12 with Stargardt's macular dystrophy. The outpatient procedure involves transplantation of retinal epithelial cells (RPE) derived from human embryonic stem cells.  RPE cells form the layer of retina just under the rods and cones, light sensitive cells that collect images to send to the brain.

The main outcome measurement of this trial is to assess safety and tolerability.  The patients receiving the stem cells have severe dry AMD and the retinal layers are destroyed.  No vision improvement is expected in this clinical trial.

The first patient is a 77 year-old woman with AMD who is legally blind.  She was injected with the smallest level (50,000 cells).  Three patients will receive this small dose and the dose will be gradually increased for later patients, up to 200,000 cells.

When this trial is completed, we will know if the procedure was safely tolerated by these 24 patients, hopefully up to the highest dose.  If that happens, a larger clinical trial will be done.  The procedure should work best if performed earlier in the vision deterioration process, so patients for that trial will be recruited before their retinas are severely damaged. In that trial, we'll learn more about possible vision improvement with this therapy.

See our previous report for more information.

July 2011