After more than 20 years of research, doctors at UCLA’s Jules Stein Eye Institute have begun treating the first patients in clinical trials for two progressive eye diseases that cause blindness: dry age-related macular degeration and Stargardt’s macular dystrophy.
The patients were given an injection of specialized eye cells that were derived from embryonic stem cells. Dr. Steven Schwartz, who is leading the trial at UCLA, performed both stem cell transplant surgeries Tuesday. The two patients are said to be recovering without complications.
According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technologies Inc., which developed the cells and is sponsoring the trials, “you could feel the excitement in the air and that history was being made.”
The Food and Drug Administration in November approved the company’s plans to test cells created from human embryonic stem cells on 12 patients suffering from each condition. Each patient will undergo a procedure in which between 50,000 to 200,000 retinal pigmented epithelial cells created from human embryonic stem cells will be injected into their eyes.” Investigators “hope the cells will replace those ravaged by the diseases.” Previous research in rat models indicated that some vision was restored.

Stargardt’s is a genetic disorder and it strikes earlier in life. Patients start to notice visual changes as early as their teens and as late as their forties. There are a number of known genetic abnormalities in the photoreceptors that are toxic over time.
In this trial, what cells in the eye are you replacing with stem cells?
Advanced Cell Technologies Inc. has been able to take [embryonic] stem cells and differentiate them into highly functional retinal pigment epithelium that do everything they’re supposed to do. Our strategy of giving the eye brand-new, ready-to-go retinal pigment epithelium is designed for areas that are compromised, not for the areas that are gone. So we need to catch it early enough for this treatment to work.
Will the patients regain vision?
The patients’ central vision is already gone. Not rescuable. So the patients we’re enrolling in this trial know they will not be getting their central vision back.
If not to restore vision, what is the goal?
This is a safety trial. It’s not designed to improve vision. It may; and if we see a signal, that would be great news and we’re hoping we will. It’s plausible biologically, but that’s not what we’re looking for.
What results are you hoping to see?
I hope what happens is that we find this is safe and that we can optimize the dosing, and that allows us to move into eyes that are earlier in disease. That could have a real visual upside.
How long is the surgery?
Under an hour. It’s an outpatient procedure done with local anesthesia. It’s a surgery that we’ve done before – not with the injection of these stem cells, but we’ve accessed the eye before, and that’s one of the things that I’ve had a lot to do with surgically.
What does this trial mean for medicine?
We’re super-privileged to be taking this first step. It’s the unknown. These patients are doing a service for mankind. It’s inspirational.
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