Stem cell trial offers hope for vision patients

WASHINGTON Mon Nov 22, 2010 6:19pm EST

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WASHINGTON (Reuters) - Christopher Goodrich of Portland, Oregon, can't wait to stick a needle in his eye.

Goodrich hopes to be one of the first patients enrolled in clinical trial that just got a go-ahead from the U.S. Food and Drug Administration, only the second trial approved anywhere in the world to test human embryonic stem cells in people.

Advanced Cell Technology, a small Massachusetts-based biotechnology company that has struggled to bring human embryonic stem cells from the lab to the clinic, will begin recruiting 12 patients in Oregon and Massachusetts with a rare eye condition called Stargardt's macular dystrophy.

Goodrich, 55, retired last year from his job as coordinator for veteran's services at Portland State University when his Stargardt's macular dystrophy suddenly and inexplicably worsened.

Like most patients, Goodrich had seen his vision get steadily worse since his young adult years.

"Glasses help very, very little," Goodrich said in a telephone interview.

"What macular degeneration is, you know that that center part of your eye that does all the fine tuning? The cells die. You know what a shotgun blast looks like? That is what I see. It's all over the place. I might see something out of the corner of my eye and turn my head and then I can't see it."

It is infuriating and Goodrich would do anything to get better, even allow his eye doctor to poke a needle into his eye and infuse human embryonic stem cells that ACT hopes will take up residence and replace the missing cells.

"I've made a list of all the things I would love to be able to do and all the things I would love to be able to see," Goodrich says.

OTHER EYE DISEASE IN SIGHTS

This is the most immediate promise of human embryonic stem cell research -- that these immature cells can be coaxed partly down the road to becoming a desired tissue type, and in the process get other cells growing and renewing.

Advanced Cell Technology's product is a human embryonic stem cell trained to become a retinal cell.

For this first clinical trial, only a few thousand of these cells will be injected into one eye of each volunteer, mostly to check to see that it is safe.

ACT's Dr. Robert Lanza says the doctors taking part in the trial will be able to watch it unfolding in real-time, because it is possible to look into the eye and see the cells taking up residence in the retina.

And it will be possible to continuously check the vision of the patients, to see if it is improving.

Stargardt's affects only 30,000 to 50,000 Americans, according to the American Macular Degeneration Foundation, but ACT has its sights set on a much larger market if this trial is successful -- age-related macular degeneration.

This blinding condition affects more than 30 million people worldwide and is the leading cause of blindness in people over 60 in the United States.

Goodrich has been a patient of Dr. Peter Francis of Oregon Health & Science University, who will be one of the investigators on the ACT trial. But Lanza said all patients will have to undergo an approval process.

(Editing by Cynthia Osterman)

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