Scientists are reporting the first strong evidence that human embryonic stem cells may be helping patients.
The cells appear to have improved the vision in more than half of the 18 patients who had become legally blind because of two progressive, currently incurable eye diseases.
The researchers stress that the findings must be considered preliminary because the number of patients treated was relatively small and they have only been followed for an average of less than two years.
But the findings are quite promising. The patients had lost so much vision that there was no expectation that they could benefit, the researchers say.
"I'm astonished that this is working in the way that it is — or seems to be working," says Steven Schwartz, a UCLA eye specialist who led the study, which was published Tuesday in the British medical journal The Lancet. "I'm very excited about it."
Other researchers agreed the work is preliminary, but also highly promising.
"It really does show for the very first time that patients can, in fact, benefit from the therapy," says Dr. Anthony Atala, a surgeon and director of the Wake Forest Institute for Regenerative Medicine at Wake Forest University.
"That allows you to say, 'OK, now that these cells have been used for patients who have blindness, maybe we can also use these cells for many other conditions as well, including heart disease, lung disease and other medical conditions,' " Atala says.
Human embryonic stem cells have the ability to become any kind of cell in the body. So scientists have been hoping the cells could be used to treat many diseases, including Alzheimer's, diabetes and paralysis. But the study is the first human embryonic stem cell trial approved by the Food and Drug Administration that has produced any results.
"It is really a very important paper," Atala says.
The study involved patients suffering from age-related macular degeneration and Stargardt's macular dystrophy, the two leading causes of adult and juvenile blindness in the developed world, Schwartz says. The diseases destroy a person's central vision.
"Whatever you're looking at is gone — whether it's faces, or reading or food on a plate, or whether something is a step or stripe," Schwartz says. "It's very, very difficult to perform activities of daily life that we, you know, don't even think about."
Working with Advanced Cell Technology Inc. of Marlborough, Mass., Schwartz and his colleagues took human embryonic stem cells and turned them into the kind of cells that are killed by these diseases — retinal pigment epithelial cells. Then, they infused between 50,000 and 150,000 cells into the retinas of the patients.
"What we did is put them into patients who have a disease where those particular cells are dying; and we replaced those dying tissues with new tissue that's derived from these stem cells," Schwartz said. "In a way it's a retinal transplant."
No one expected the cells to help any of these patients see better, because the study was designed mostly just to see if doing this was safe. Researchers were concerned the cells could destroy whatever vision was left or lead to tumors in the volunteers' eyes. So Schwartz picked patients whose eyes were so far gone that they weren't risking losing any vision. That also meant that there was little hope the cells could help either.
"We did not expect to help these patients, and they did not expect to be helped," Schwartz says.
Some patients experienced side effects from the procedure itself and from the drugs they had to take to suppress the immune system, but none of the side effects were considered serious. The cells themselves have produced no safety problems so far, the researchers reported.
And, surprisingly, many of the patients did start to see better, according to the report. Ten of the 18 patients can see significantly better. One got worse, but the other seven either got better or didn't lose any more vision.
"These are patients that didn't see better for 30 years and all of a sudden they're seeing better," Schwartz says. "It's amazing."
The patients include a graphic artist who could suddenly make out the woodwork carved on a piece of furniture in her bedroom, an international consultant who regained the ability to walk through busy airports without help, and an elderly rancher who's riding his horse again, Schwartz says.
"He couldn't see things like a barbed-wire fence or whether in the distance a stray cow was under a tree," Schwartz says. "And six months after the transplant he's back to running his cattle again. And he can, in fact, see a snake on the ground or sort of tell whether a distant shadow is a cow or something else. So it's made a big difference for him in his life."
Isabella Beukes of Santa Rosa, Calif., has been legally blind for more than 40 years. But within weeks of getting the cells, she started to see better. She could make out the cursor on her computer screen and the color of her clothes. Today, she can hike the hills near her house all by herself.
"The improvement, I mean, from where I was coming is just, it's very, very significant for me. I think it's fantastic," Beukes says. "I just think to be part of groundbreaking research work is amazing."
The research is controversial, however. Embryos are destroyed to get the cells, and some people think that's immoral.
"The problem we have with embryonic stem cells is simply the fact that you have to destroy a young human being to get embryonic stem cells," says David Prentice, senior fellow for life science at the Family Research Council, an advocacy group. "We would reject the idea that any human being be destroyed for experimental purposes."
For his part, Schwartz says he's just trying to help blind people see better. But he cautions that this work is still at a very early stage.
"I don't want patients to come in to their doctor saying, 'Hey, I heard about the stem cells on the radio and I'd really like to get that treatment done, and what do you think?' " he says. "It's not ready. Maybe in a few years. Maybe not. We have to wait and see. The jury is way out still."
Schwartz has continued treating more patients using larger doses of cells and trying it on patients who haven't lost as much vision to see if that works even better. He has also expanded his study to Boston, Miami, Philadelphia and London.
STEVE INSKEEP, HOST:
The use of embryonic stem cells is changing. It's going from science that could someday help patients, to treatments that actually do. Scientists are reporting the first strong evidence that human embryonic stem cells can help patients. In a paper in the scientific journal The Lancet, researchers report that cells seem to have enabled some people blinded by eye diseases to see better. It's a promising treatment and also controversial. NPR's Rob Stein takes us inside the operating room.
ROB STEIN, BYLINE: It's early on a Tuesday morning at the UCLA Jules Stein Eye Institute in Los Angeles, and an elderly woman is getting ready to try something daring.
JOYCE: My name is Joyce, and I live in Lincoln, outside of Sacramento. And do I have to give you my age of 80? I just turned 80.
STEIN: After we talk a bit more, Joyce decides she's OK with the age thing. But she doesn't want us to use her last name. She knows she's getting involved in something that some people think is morally wrong.
JOYCE: There are some people, because of their religious background, are a little bit hesitant at the use of stem cells.
STEIN: And Joyce is afraid of being targeted by activists who object to this kind of research. But she hopes she can help scientists learn how to use human embryonic stem cells. She has macular degeneration, the most common cause of blindness in older people.
JOYCE: I have very low vision. I have struggled with it for several years.
STEIN: She's not totally blind, but she can't see much. So she's had to give up all kinds of things - going shopping by herself, working at her church. But the hardest part is not seeing faces, especially her grandchildren. She tears up just talking about them.
JOYCE: I have five. I know, you know, what they look like. I know - the three older children, I know very well what they look like. It's just that I would like to see them more, and they change. We have a new baby in the family that's only 5 months old, so I don't see her as well as I'd like to.
STEIN: So Joyce volunteered for a study to see if human embryonic stem cells could help her or at least help other people someday.
JOYCE: When I heard there would be any chance at all, I felt even if it didn't help me, I still wanted to participate.
STEIN: Human embryonic stem cells can turn into any kind of tissue in the body. So ever since they were discovered, scientists have thought that one day they might be able to cure lots of diseases. The study Joyce is volunteering for is the first that's getting a glimpse of whether that might be true. Later that day, Joyce is lying on an operating table surrounded by doctors, nurses and technicians. She checks with the anesthesiologist to make sure she'll be out of it during the procedure.
JOYCE: I want you to know what you're doing, but I don't want to know what you're doing.
UNIDENTIFIED WOMAN: Sounds good. Sounds like a plan.
STEIN: Once everything is ready, Dr. Steven Schwartz, the eye surgeon running the study, gets started.
STEVEN SCHWARTZ: Lights out, please.
WOMAN: Lights down.
STEIN: With the room darkened, Schwartz peers into Joyce's eye through a big microscope hovering just above her face at the end of a mechanical arm. He delicately inserts three tiny tubes into Joyce's eye, including one he'll use to infuse cells into her damaged retina, cells that were made in a laboratory from human embryonic stem cells - 200,000 cells called retinal pigment epithelial cells.
SCHWARTZ: Can I have the cells, please?
STEIN: An assistant pulls vials containing the cells from a small, red cooler so Schwartz can load them into a special syringe.
SCHWARTZ: OK, perfect.
STEIN: He inserts the tip of the syringe into one of the tiny tubes in Joyce's eye and squeezes the plunger.
SCHWARTZ: Keep going. Beautiful. Beautiful.
STEIN: The cells are right where he hopes they'll take root, grow and possibly regenerate dead parts of Joyce's retina. After about 30 minutes, the procedure is done, and they wheel Joyce into a recovery room. Afterwards, Dr. Schwartz tells us it will take weeks, maybe months, to see what the cells are doing.
SCHWARTZ: Our hope is that she will be safe and perhaps contribute to our understanding of optimizing this procedure for future patients and maybe, in our wildest dreams, glean a benefit personally from this.
STEIN: Wildest dreams because the study was designed just to see if it's safe. The fear was that the cells would end up destroying whatever vision's left or morph into grotesque tumors. So Schwartz started with patients whose eyes were so far gone, there was little chance it would help or hurt.
SCHWARTZ: We don't want to take a sighted eye or an eye that has really meaningful vision and blind it, which we could well have done.
STEIN: So far, there are no signs the cells are dangerous. More than two dozen patients have been treated, and some have been followed for almost two years. And something seems to be happening that Schwartz can hardly believe. In the new study, Schwartz is reporting that more than half of the first 18 patients have started to see better.
SCHWARTZ: I'm astonished that this is working in the way that it is or seems to be working, and I'm very excited about it.
STEIN: One volunteer got worse, but 10 could see a lot better. Seven others either could see a little better, or at least didn't lose any more sight. And for some, Schwartz says, it's been dramatic, like an elderly rancher who lives in the Midwest.
SCHWARTZ: He came in at his six-month visit, took his hat off, took his glasses off and started crying in my office. And he said, you know, I don't cry. I've never cried, and I can't tell you, doc, how much this means to me that I can take my horse out and ride my cattle and be on the land and be productive and be independent. That was pretty emotional.
STEIN: One of the first patients to volunteer for Schwartz's study was Isabella Beukes. Beukes is out for a hike in a nature preserve near her house in Santa Rosa, California. Before she got the stem cell treatment two years ago, this would have been impossible.
ISABELLA BEUKES: In the past, it was hazardous, you know. I could fall.
STEIN: Just walking anywhere by herself has been hard for Beukes for almost 40 years. She's 56 and lost most of her vision when she was a teenager because of a condition called Stargardt macular dystrophy. But within weeks of getting the cells...
BEUKES: All of a sudden, things happened. And they couldn't believe it. And then my children were so used to walk with me, you know, to say, mind the step, mind this. And my daughter was with me and in Los Angeles, and so she just burst into tears because she couldn't believe - what I could see.
STEIN: Beukes could make out the color of her clothes, see the cursor on her computer screen. Slowly, she realized she could find her way through crowded restaurants and even busy airports by herself. And today, she's hiking steep, rocky paths in a nature reserve.
BEUKES: I'm not scared of tripping anymore because, you know, I can actually see where I'm going. You see that, for instance, that stick that was lying there in front of us now? I could see it and avoid it, where in the past, I would basically trip over it.
STEIN: And when she looks into the distance, she can see the giraffes and other animals wandering the hills.
BEUKES: And I can see, for instance, these birds and stuff here which I wouldn't have been able to see before. I mean, I can see the trees better, for instance, and I can see, you know - for instance, I can see the flamingos and stuff like that which is - which is different. It's more clear.
STEIN: Now, Dr. Schwartz and others caution that the results still have to be considered very preliminary. After all, he's still only treated a relatively small number of patients.
SCHWARTZ: I don't want patients to come into their doctor and say, hey, I heard about the stem cells on the radio, and I'd really like to get that treatment done, and what do you think? It's not ready. Maybe in a few years. Maybe not. We have to wait and see. The jury is way out still.
STEIN: In the meantime, Dr. Schwartz is continuing to treat more patients like Joyce, the patient we heard about at the beginning of our story. She's still waiting to see what her cells are doing. Schwartz has also expanded his study to Boston, Philadelphia, Miami and London. And doctors are trying the cells on patients who haven't lost as much of their vision in the hopes that human embryonic stem cells may prevent people from going blind in the first place. Rob Stein, NPR News. Transcript provided by NPR, Copyright NPR.