Embryonic Stem Cells Restore Vision In Preliminary Human Test
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.