靶向抗癌药物给骨髓瘤患者带来新生

LINDA WERTHEIMER, HOST: This is MORNING EDITION from NPR News. I'm Linda Wertheimer.

RENEE MONTAGNE, HOST: And I'm Renee Montagne. Good morning.

Today in Your Health, we have two stories about cancer. The first about what was once one of the most fearsome cancer diagnoses: multiple myeloma.

But NPR's Richard Knox reports on the remarkable progress in treating that disease.

RICHARD KNOX, BYLINE: Not so long ago, a diagnosis of myeloma meant you'd have a short and painful future. This blood cell cancer would literally eat holes in your bones. They could break just by bending over to pick up a package.

That's no longer true for most of the 20,000 Americans who get myeloma every year and thousands more who are living with it.

DONALD WRIGHT: My name is Donald Wright - Don Wright. And I live in Lake Elmo, Minnesota.

KNOX: Wright got diagnosed with myeloma at what turned out to be the right time.

WRIGHT: That was in 2003. That was going on 10 years ago. I was 62, I guess.

KNOX: That was right after he finished running his first marathon. But the cancer hasn't stopped him. Over the last decade, he's averaged seven marathons a year. He's training for his 71st next month on Cape Cod.

WRIGHT: Yesterday, I ran 18 miles and then came back and shoveled snow for two-and-a-half hours. That was a big day.

KNOX: Wright's remarkable health is due to a drug called pomalidomide. It's a chemical cousin of thalidomide, a drug that caused birth defects back in the early '60s. This month, the Food and Drug Administration approved pomalidomide for myeloma. It'll be sold as Pomalyst.

Don Wright was one of the first patients to get it, back in 2006.

WRIGHT: For me, this has been a miracle drug. It's kept the myeloma stable. And it's just a little pill. You know, I take that little pill every night. I take it the night before I run a marathon and the night after I run a marathon. It barely makes a difference in my lifestyle.

KNOX: Pomalyst is the latest of a half-dozen myeloma drugs developed since Wright got diagnosed. Another, called Kyprolis, won approval last summer.

Dr. Brian Durie of the International Myeloma Foundation says these drugs have been transformative.

DR. BRIAN DURIE: It really has changed the whole landscape for both the doctor and the patient. Ten, 15 years ago, it was very depressing to see a new myeloma patient because you knew that the life was potentially short. And it was difficult to be overly optimistic about what might transpire.

KNOX: But now, doctors can promise something pretty remarkable.

DURIE: We can say 95 to 98 percent of the time we can pretty much guarantee that we will have a treatment that works, that the patient will go into remission.

KNOX: This is not a cure. Eventually the first drug regimen, whatever it is, will stop working and the cancer comes back. But researchers think they're on the verge of blocking the cancer's return for good.

Here's Dr. Ken Anderson of the Dana Farber Cancer Institute in Boston.

DR. KEN ANDERSON: We're close, close, close. We're soon really going to change the natural history in a major way.

KNOX: That's doctor-speak for cure. Until specialists achieve that cure, they have an expanding bag of tricks to keep the cancer at bay; new drug combinations and more new drugs coming along.

ANDERSON: If somebody comes and sees us in the clinic, you can look them in the eye and say, you know, it's highly likely that you're going to live a decade or more. And, frankly, with the maintenance therapy, it could be quite a lot longer than that as well.

KNOX: Marathoner Don Wright has his own definition of a cure.

WRIGHT: The cure for myeloma is to hang on long enough to die of something else. And that is precisely my hope and my plan, actually.

KNOX: You might say he's in it for the long run.

Richard Knox, NPR News