A QUESTIONABLE CURE

May 13, 1999
		Bone marrow transplants have been giving hope to people with stubborn cases of breast cancer for years, but the effectiveness of the procedure has never been clear. A new study has renewed the debate. The Health Unit is a partnership with the Henry J. Kaiser Family Foundation.

Feb. 18, 1999: Preventative Mastectomies Am I at risk? Sept. 25, 1998: The Cancer March May 27, 1998: Sorting out cancer research April 13, 1998: New drugs to treat breast cancer March 18, 1998: Is Vitamin E a cancer fighter? March 12, 1998: Some cancers are declining. May 30, 1996: Lawsuits against breast implant manufacturers. Nov. 25, 1996: More news about Prostate Cancer More NewsHour Health and Science coverage.	SUSAN DENTZER: Nine of Joanne Ruddy's eleven children gathered in a hospital room last month to wish their mother a happy 55th birthday. KIDS SINGING: Happy Birthday, Dear Mom, Happy Birthday to you.
	Unproved treatment?
	SUSAN DENTZER: As she opened gifts and read cards from her two absent children, Joanne seemed eager to return home from Georgetown University Hospital after her latest bout of treatment for breast cancer. JOANNE RUDDY: Thank you everybody -- a lot to look forward to when I get home. SUSAN DENTZER: It's been almost a year since Joanne, a former school teacher, was diagnosed with inflammatory breast cancer. That is one of the most aggressive and least curable forms of the disease. JOANNE RUDDY: "Dear Mom, everyone who still thinks you look terrific raise your hand"-- SUSAN DENTZER: Once the cancer was discovered, Joanne had chemotherapy to kill cancer cells that may have spread throughout her body. That was followed by a mastectomy and still more chemotherapy. Then, even as she celebrated her birthday, she was undergoing still another treatment -- doses of chemotherapy so powerful that they virtually destroyed her bone marrow, blood supply, and immune system. To keep Joanne from dying from the high-dose chemotherapy, special cells known as "stem cells" had been removed from her blood beforehand. They were mixed with a preservative and temporarily frozen. When the high-dose chemotherapy was complete, the cells were thawed. Then they were transfused back into Joanne Ruddy's body to help her bone marrow, blood supply and immune system regenerate. HEALTH CARE WORKER: And then we're just going to do your vitals every fifteen minutes for one hour. SUSAN DENTZER: Just days before Joanne Ruddy underwent the treatment last month, this same therapy made the news. Splashed across the front pages of the nation's leading newspapers were headlines like these. The stories reported on five major studies of breast cancer patients who had undergone similar high-dose chemotherapy and transfusions, known as "transplants". At first glance, most of the studies seemed to show little, if any, benefit from the procedure. Joanne Ruddy wasn't deterred. JOANNE RUDDY: My doctor all along has told me that they don't have definitive answers, that they don't have enough information yet. I'm putting my trust in God that, you know, you have to take a chance. DR. KENNETH MEEHAN: Hi! How's it going, okay? JOANNE RUDDY: Yes -- SUSAN DENTZER: Joanne's physician, Dr. Kenneth Meehan, is a breast cancer transplant specialist at Georgetown.
	A last resort.
	DR. KENNETH MEEHAN: Most patients, in general, when they come to see me, they will do anything to try to live as long as they can without disease. Joanne, in particular, has a very large family, a number of children. She wanted to be very aggressive. SUSAN DENTZER: That determination has hurtled Joanne into the midst of a raging medical controversy: How well -- and for which breast cancer patients -- do high-dose chemotherapy and transplants work? The debate constitutes a case study of the most difficult issues in medicine, where costly innovations offer new hope to the very sick -- however slim that hope may be. Dr. Lee Newcomer is medical director of United Health Group, one of the nation's largest health maintenance organizations. A cancer specialist, he has reviewed hundreds of cases of patients seeking high-dose chemotherapy and transplants. DR. LEE NEWCOMER: I think the big issue is what's the right thing to do, and do we have some science and some evidence to tell a woman facing this decision what she really has to look forward to in terms of side effects, and in terms of outlook. SUSAN DENTZER: A woman, that is, like Joanne Ruddy -- or like 49-year-old Sandra Rolef, who had a mastectomy for breast cancer several months ago. By that time, the disease had spread to 16 of her underarm lymph nodes, putting her at very high risk for a recurrence. Along with her husband, Rolef is now consulting oncologist, Dr. Robert Siegel of George Washington University. They are considering whether to go ahead with the high-dose chemotherapy and stem cell transplant. DR. ROBERT SIEGEL: It's not a slam dunk one way or the other. SANDRA ROLEF: So you're not ready to really make a final recommendation to me yet? DR. ROBERT SIEGEL: I just think it's important not to jump to conclusions before you have to. SUSAN DENTZER: Although Dr. Siegel is uncertain, other doctors Rolef consulted encouraged her to proceed.
	"It's a kind of personal decision."
	SANDRA ROLEF: One of the doctors said something to me that really is kind of sticking in the back of my mind, and she said, "You know, it's kind of a personal decision. And if you're the kind of person that wants to make sure that no stone is unturned, that you have done everything humanly possible to fight this disease and make sure that you're rid of it, then you should do it." SUSAN DENTZER: The debate over the treatment's effectiveness has raged since the 1970s. Back then, high-dose chemotherapy and transplants were first used successfully to treat other forms of cancer, such as leukemia. Inevitably, doctors also began testing the approach in "advanced" cases of breast cancer in which the disease had spread to organs or bones. Such patients were given doses of chemotherapy drugs that were five to 30 times higher than those used in conventional treatment. DR. LEE NEWCOMER: There was a lot of theory about the bigger the dose, the better the chance of getting rid of the cancer. By the middle 1980s this was a fairly common procedure. SUSAN DENTZER: At the time, doctors used patients' own bone marrow for the transplants. They later switched to using blood stem cells when these proved just as effective. The treatment was costly -- as much as $200,000, or several times the price of more conventional breast cancer therapy. It was also very risky. Early on, as many as 1 out of 5 who got the treatment died from it, rather than from breast cancer. Besides the risks, there was also no hard scientific evidence that the treatment was effective. As a result, many health insurers balked at paying the costs. Breast cancer survivor Fran Visco is president of the National Breast Cancer Coalition, a group of 25 patient advocacy organizations. FRAN VISCO: Physicians would say to women, a bone marrow transplant is the only thing that could possibly save your life. And you had situations where women sued insurance companies, requiring their company to cover a bone marrow transplant. SUSAN DENTZER: The quest for treatment generated some of the highest-profile lawsuits brought against health insurers over the past decade. One involved this California woman, who sued her HMO, got the treatment, and subsequently died. In her case, as in others, courts ordered insurers to pay the costs. FRAN VISCO: Well, insurance companies threw in the towel and started paying for bone marrow transplants broadly because of some of the verdicts.
	But does it work?
	SUSAN DENTZER: But there was still little hard evidence that the treatment worked -- the kind of information only gained from rigorous clinical trials. That's in part because assembling such studies was difficult. In a well-done clinical trial of this type, patients are randomly assigned to receive either the experimental treatment being tested or the standard, effective therapy. But many patients resisted entering trials, since they were convinced that the experimental treatment was their only hope. DR. KENNETH MEEHAN: Patients nowadays are very intelligent. They do not want to be randomized to the chemotherapy line, despite my emphasis saying, 'We're not sure if this works at this point in certain situations. I would recommend this trial.' They would go elsewhere. SUSAN DENTZER: Out of the estimated 12,000 women who underwent the treatment, only 1,000 participated in clinical trials. FRAN VISCO: The real tragedy in this story is that if women had enrolled in the bone marrow transplant trials, if their physicians had encouraged them to do so, we would have had the answer years ago, and it would have saved lives. SUSAN DENTZER: Gradually, though, major trials were assembled and began to accumulate results. One conducted from 1990 to 1997 tracked women with advanced cancer that had spread to organs or bones. After three years, the study showed no difference in survival rates between patients who got the new therapy and those who got the standard treatment. When this and other major studies made news last month, Sandra Rolef says -- SANDRA ROLEF: It had my telephone ringing -- lots of friends and family hysterical over it because of what the studies say. 'Why are you doing this, and, you know, why don't you just take your stem cells and freeze them and think about it?'. SUSAN DENTZER: But the studies have not cleared up the controversy and they may actually have deepened it. On one side, Fran Visco's group argued that the procedure was fruitless. FRAN VISCO: High-dose chemotherapy with bone marrow transplants for breast cancer is not an effective therapy.
	Clearing up the controversy.
	SUSAN DENTZER: But other cancer specialists argue that the results of the studies were far more ambiguous. Dr. John Durant is executive vice president of the American Society of Clinical Oncologists, known as ASCO. DR. JOHN DURANT: It's way premature to say this strategy doesn't work. I think the strategy will continue to be of interest as a means of improving survival. SUSAN DENTZER: To bolster their case that the jury on transplants isn't yet in, Durant and other specialists point to the complexities of the studies. One important factor is just how sick were the patients in the clinical trials. For example, the studies suggested the new therapy did not improve survival of patients with advanced cancer that had spread to organs or bones. On the other hand, the treatment looked more promising for patients like Sandra Rolef, whose cancer had only spread to 10 or more underarm lymph nodes. In one study conducted in South Africa, these high-risk patients who underwent the experimental therapy were far more likely to survive. Some of the clinical trials will now continue, and in addition, doctors say new studies are needed. For example, they want to know how effective the treatment is for other forms of breast cancer, such as the inflammatory cancer that afflicts Joanne Ruddy. They also want to understand the effects of treatment innovations that have taken place since the first studies began. For example, new procedures have cut the transplant death rate to as low as 1 in 20 patients. And powerful new chemotherapy drugs such as Taxol may also have yielded better results. DR. KENNETH MEEHAN: I think that in the long run there will be a small advantage to transplant. It's not going to be 50, 60, 70 percent. It may be 10 percent, maybe a little bit higher. SUSAN DENTZER: But Durant says it will be a while before those results are in. SUSAN DENTZER: On a scale of one to ten, one being we know almost nothing about all of this, ten being that we know everything, where are we? DR. JOHN DURANT: Oh, we're at two or three probably. SUSAN DENTZER: The uncertainty raises the stakes for patients like Sandra Rolef. SANDRA ROLEF: I don't want to look back in a year, or two years, or five years and say I should have, when they have more data, and maybe the data is going to end up saying that it does help. And how could I live with myself? SUSAN DENTZER: After all, for Rolef, Ruddy and their families, the goal is living to celebrate more birthdays. KIDS: All right! clapping SUSAN DENTZER: The transplant studies will be a major topic of discussion next week. That's when cancer experts meet in Atlanta for ASCO's annual conference.