First Hints That Stem Cells Can Help Patients Get Better : Shots - Health News Sue Freeman, 78, is one of two women who appear to have unexpectedly regained some of their vision while taking part in a study testing the safety of an embryonic stem-cell therapy. The findings, if confirmed, could mark a landmark step in stem cell therapy, but everyone involved in the work is being very cautious about how they describe these results.
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First Hints That Stem Cells Can Help Patients Get Better

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First Hints That Stem Cells Can Help Patients Get Better

First Hints That Stem Cells Can Help Patients Get Better

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Human embryonic stem cells have generated a lot of excitement and a lot of controversy, but no results yet - at least not in human patients. Well, scientists are now reporting the first hints that the cells may have helped someone get better. Two women appear to have unexpectedly regained some vision while volunteering for a preliminary study.

As NPR's Rob Stein reports, everyone involved in the work is being extremely cautious about how they interpret the results so far.

ROB STEIN, BYLINE: The provocative news is about two patients with progressive, incurable eye diseases. Steven Schwartz of UCLA is leading the research and describes their conditions this way.

DR. STEVEN SCHWARTZ: If you wanted to imagine what one of these patients was going through, you could hold the palm of your hand about one inch from your nose and look straight ahead. You'd see a huge blind spot. If you look down to the right or the left, the palm or your hand or the blind spot would move with you. So wherever you look, you can't see.

STEIN: Eventually, patients with macular degeneration often lose their ability to read, recognize faces, drive, work, even go outside on their own. Now, the main goal of this study is really just to see whether it's safe to inject cells made from human embryonic stem cells into someone's eye.

SCHWARTZ: Imagine sitting there with your doctor and he tells you that we don't know whether or not this is going to help you or this is going to hurt you. We don't know whether or not these stem cells are going to turn into some sort of tumor, or other problem, or whether it's going to remove the remaining vision that you have in that eye.

STEIN: Even with those warnings, two of Schwartz's patients agreed to let him inject 50,000 cells into one of their eyes in July. Both women had lost so much vision and the dose they got was so low that no one expected the cells would actually help them see better. But within weeks, both started to think something might be happening.

UNIDENTIFIED WOMAN: I sort of like woke up one morning and did realize that, wow, you know, there is a difference between the two eyes now. They only worked on the left eye. The furniture on the other side of the room has a lot of - I have some hand-carved furniture there, and I could actually see the detail on the carving. You know, on the other side of the room there, on things that I couldn't see from that distance before.

STEIN: This patient had started going blind in her 20s because of a disease called Stargardt's macular dystrophy. She's in her 50s and lives in Los Angeles. She didn't want her name used because she's worried about losing work. It turns out, she's a graphic artist.

UNIDENTIFIED WOMAN: It was pretty amazing. I mean, I was like kind of looking at everything new again, you know.


UNIDENTIFIED WOMAN: Just sort of going around and first not believing it, but then really looking, you know. And realizing that, you know, I definitely had more sight in that eye.

STEIN: The other patient is Sue Freeman of Laguna Beach, California. She has a condition known as dry, age-related macular degeneration. It's the leading cause of blindness in the developed world. The disease had slowly destroyed Freeman's ability to read, drive, cook, and do so many things that were once so easy. It got to the point where she couldn't even go outside for a walk by herself.

SUE FREEMAN: Everything got harder. You know, simple things like seeing a telephone, turning on a TV, pouring a glass of water without spilling it

STEIN: But within weeks of getting the cells, Freeman, who's 78, noticed landscapes seemed clearer and brighter. Soon, she was making her own breakfast again. One day, she felt so much better she convinced her husband to drop her off at the mall so she could go shopping.

FREEMAN: One day I looked down and I could see my watch, which I wear it even though, because I like jewelry. So, I always wear it, but I probably hadn't seen it in about a year and a half or two. And I could see. That was exciting for me. And I remember saying: Oh, my goodness. I can see my watch. I can actually tell time.

STEIN: UCLA's Steven Schwartz is pretty confident that the graphic artist might really have gotten better because of the cells. When his team examines her treated eye, they can actually see the transplanted cells thriving. He's a little less certain about Sue Freeman. At first, he suspected the placebo effect, and he's worried about raising expectations too high, too fast.

SCHWARTZ: And my job is to decrease suffering. And if we overstate this and raise hopes falsely and then it doesn't work out, it will hurt people rather than help them.

STEIN: The company that made the cells, Advanced Cell Technology, has the OK to treat a total of 24 patients in the United States and 12 in the United Kingdom. Clearly, they have a lot more work to do to make sure the cells are safe, let alone establish that they are really working. After all, the results they have so far come from just two patients.

Schwartz plans to treat a third patient on Tuesday. And doctors in London have started injecting cells into patients there last week.

Rob Stein, NPR News.



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