Two women losing their sight to progressive forms of blindness may have regained some vision while participating in an experiment testing a treatment made from human embryonic stem cells, researchers reported today.
The report marks the first time that scientists have produced direct evidence that human embryonic stem cells may have helped a patient. The cells had only previously been tested in the laboratory or in animals.
"I can't tell you how excited I am about this," said Steven D. Schwartz, a professor of ophthalmalogy at UCLA's Jules Stein Eye Institute leading the research. "For these patients, the impact is enormous."
Schwartz and his colleagues stressed that the findings are extremely preliminary and it's far too early to know anything for sure. The patients could continue to improve, or their vision could deteriorate again, he said. Many more patients will be needed to be treated for far longer to know whether the therapy is really safe and responsible for any improvement.
"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," Schwartz said.
But the findings could mark an important milestone for the field of human embryonic stem cells, and so-called regenerative medicine, Schwartz and others said.
"In the landmark paper by Schwartz and colleagues, the potential to use human embryonic derived cells with a therapeutic effect in patients is now finally realized," wrote Anthony Atala of the Wake Forest Insitute of the Wake Forest School of Medicine in a commentary accompanying the report in the journal The Lancet.
Scientists discovered human embryonic stem cells in 1998. Many researchers believe they could revolutionize medicine because they can morph into virtually any type of cell in the body. That means they could potentially provide cells to treat many diseases, including diabetes, Alzheimer's, Parkinson's and spinal cord injuries.
But the field has been the focus of intense debate and controversy because human embryos are often destroyed to obtain the cells. Critics consider any research on human embryos, and especially the destruction of human embryos, to be immoral.
The Food and Drug Administration has only approved two studies testing any therapies made from human embryonic stem cells in people. The first involved patients who were partially paralyzed by spinal cord injuries. But that experiment was discontinued soon after it began last year when Geron, the company sponsoring the research, announced it was diverting funds to other projects.
The blindness study, which is being sponsored by Advanced Cell Technology, will involve 24 patients suffering from Stargardt's macular dystrophy, which is the leading cause of pediatric blindness, and dry age-related macular degeneration, which is the leading cause of blindness in the developed world.
Scientists used embryonic stem cells to create retinal pigment epithelium cells in the laboratory. RPE cells are lost in a variety of eye conditions, including Stargardt's and macular degeneration.
In July, Schwartz and his colleagues injected about 50,000 RPE cells made from stem cells into the right eye of Sue Freeman, 78, of Laguna Beach, Calif., who had lost most of her vision to macular degeneration.
Because of the disease, she could no longer recognize faces, read, cook or even go outside on her own. But within six weeks of the procedure, Freeman started to notice she could see landscapes better. Tests showed she could read more letters on an eye chart. Soon, she was making her own breakfast again and even has gone shopping alone.
"One day, I looked down and I could see my watch," she said. "I probably hadn't seen it in about a year and a half or two. And I could see. So that was exciting for me. And I remember saying, 'Oh my goodness. I can see my watch. I can actually tell time.' "
Schwartz and his colleagues are somewhat baffled by Freeman's improvement, especially since she initially reported being able to see better with both eyes, including the one that wasn't treated.
That made them suspect that something else may be causing the seeming improvement, such as the anti-rejection drugs she was taking or the placebo effect. But they have since become more confident her improvement is indeed being caused by the cells.
They are even more confident about the improvement by the second patient, who asked not to be identified to protect her privacy. She's a woman in her 50s who works as a graphic artist and started losing her sight in her 20s because of Stargardt's. When doctors examine her eye, they can actually see the transplanted cells thriving.
"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," she said. "On the other side of the room 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."
She has since begun working much more easily, doing routine chores around the house and even riding a bike again.
"It was pretty amazing. I was like kind of looking at everything new again, just sort of going around and first not believing it but then really looking and, you know, realizing that I definitely had more sight in that eye," she said.
Now, Schwartz and his colleagues stress that the study was designed primarily to determine whether the cells were safe, and not to determine whether they might actually help patients. For this study, patients received very low doses, and their vision had been so damaged that no one thought the cells could help them.
Schwartz treated a third patient on Tuesday, and doctors in London began treating Stargardt's patients there on behalf of the company. Much more research is needed, they stress.
"It's just far too early to make any conclusions," said Robert Lanza, the company's chief scientific officer.
But if the findings are confirmed, Lanza said they would mark an important, long-awaited step.
"I think this is a turning point. It's been 13 years since the discovery of human embryonic stem cells," Lanza said. "We've been reading about this. It's been one of the hottest topics of biology. And this is the first report of the effects of these cells actually transplanted into a human patient. So it's been a long time in the works."
ROBERT SIEGEL, HOST:
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.
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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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SIEGEL: This is ALL THINGS CONSIDERED from NPR News. Transcript provided by NPR, Copyright NPR.