美国国家公共电台 NPR Gene Therapy Shows Promise For A Growing List Of Diseases

STEVE INSKEEP, HOST:

Now we have a story about the power of science to heal. For decades scientists have been dreaming about curing diseases with gene 1 therapy. Those dreams have not become reality, but there's growing excitement now. NPR health correspondent Rob Stein reports.

ROB STEIN, BYLINE 2: Natalie Wheatley was terrified there might be something wrong with her baby even before he was born. He barely moved during her pregnancy 3, and after he was born, doctors told her her worst fears were true. He had a terrible incurable 4 genetic 5 disease that was destroying his muscles.

NATALIE WHEATLEY: Basically told me he wouldn't make it to his first birthday, take him home and love him and spend as much time with him as you could. And I'm going to be honest with you. I was devastated 6, devastated.

STEIN: When Christian 7 Guardino was born, he was also diagnosed with a rare genetic disease. His wasn't fatal but was destroying his vision.

CHRISTIAN GUARDINO: I wouldn't be able to walk around outside by my own. It would be incredibly dark, blurry 8 and sort of watching your world fade away.

STEIN: But Wheatley's son, Eli, is still alive. He's now 3 and seems to be thriving in Lebanon, Ky.

WHEATLEY: He just started preschool in September. He goes to preschool alone. He eats in the cafeteria with all the other kids. He's doing extremely well, extremely well. It's been amazing. Truly amazing.

STEIN: And Christian Guardino, who is 17 and lives in Patchogue, N.Y., can now see things he only dreamed about.

GUARDINO: I was able to see things for the first time, like the moon. I was able to see stars for the first time, fireworks and all these amazing things I've never been able to see before.

STEIN: Eli Wheatley and Christian Guardino are among a growing number of patients whose lives are being saved or radically 9 changed by gene therapy.

DAVID WILLIAMS: This is really an important time in gene therapy.

STEIN: That's David Williams. He's a Harvard scientist who recently reported that gene therapy could cure kids suffering from yet another awful genetic disorder 10, the fatal brain disease made famous by the movie "Lorenzo's Oil."

WILLIAMS: It really shows us that we're able to harness this therapy for some pretty terrible diseases so that's just great.

STEIN: Scientists thought this would happen a long time ago, but the first attempts to save people with defective 11 genes 12 by giving them new healthy genes fizzled. And Carrie Wolinetz of the National Institutes of Health says some volunteers in early experiments ended up getting cancer. Some even died.

CARRIE WOLINETZ: And that caused a setback 13 in the field which caused a lot of concern that maybe gene therapy was not ready for prime time.

STEIN: And may never be. But scientists went back to their drawing boards to come up with better, safer ways to deliver healthy genes into people's bodies, and that finally seems to be paying off.

WOLINETZ: I think we have reached a point of maturation in the science and in some of the new approaches to gene therapy that have allowed us to make rapid advancements 14 in a fairly short period of time. So it's a very exciting time for the field.

STEIN: Now, gene therapy has still only been tested on a small number of patients for relatively 15 short periods of time. A lot more patients will have to be studied for a lot longer before anyone really knows how well it works, for how long and whether it's really safe. Another big question is the price tag. These new gene therapies cost hundreds of thousands of dollars, perhaps even a million dollars for each patient. And Peter Bach of Memorial Sloan Kettering says that's just too much.

PETER BACH: Gene therapies, we've been hearing for years that they were going to be a million dollars before we had any product. So now when they come out, everyone's like, well, we expect them to be a million dollars, simply because it's been repeated so much.

STEIN: Whatever the questions and cost, people who have been helped by gene therapy are just thrilled. Here's Christian Guardino again, the teenager who regained 16 his vision.

GUARDINO: I think that the gene therapy is a miracle, and I think I can't imagine my life without gene therapy. I can't imagine what my life would be like without it.

STEIN: Natalie Wheatley, whose son, Eli, got gene therapy says he keeps getting better.

WHEATLEY: I see progress every day. I see progress every day so that to me offers hope that, you know, gene therapy has saved his life. And I think eventually gene therapy will give the world hope. That's my hope, anyways.

STEIN: The Food and Drug Administration could approve the first gene therapy for a genetic disorder soon, the one Christian got to save his vision. Meanwhile scientists are starting to test gene therapies for a long list of diseases including many that are much more common.

Rob Stein, NPR News.