Doctors and patients were disappointed Thursday by the preliminary results of research on an aggressive treatment reserved for the most advanced breast cancer.
Early findings from four major studies found no difference in survival for patients who underwent arduous and costly bone marrow transplants instead of less rigorous therapy. However, positive preliminary results from a fifth trial raise hope that the treatment might still help some women.
"Our results suggest that there is not a major benefit to transplant over conventional therapy," says Dr. Edward Stadmauer of the University of Pennsylvania told CBS News Correspondent John Roberts.
The treatment in question uses ultrahigh doses of chemotherapy followed by a bone marrow transplant to repair the immune system destruction caused by such high-dose therapy. Thousands of women have demanded the procedure, believing it is their last chance, even though there has been no scientific proof of its effectiveness.
The preliminary results announced Thursday from long-awaited studies of 2,100 women in the United States, Scandinavia, South Africa and France, were discouraging for many doctors and patients.
Only the South African study found a benefit of the aggressive treatment: More than five years after treatment, fewer women who got the high-dose chemotherapy plus a transplant had suffered cancer relapses. Seventeen percent of the aggressively treated women had died vs. 35 percent of women who received standard therapy.
When breast cancer spreads to other organs, called metastasis, only 22 percent of patients survive five years. Some 43,000 U.S. women will die of it this year. Given such statistics, many patients have demanded the ultrahigh dose therapy and bone marrow transplant, which doctors refer to as HDC/BMT.
Some patients have found the treatment successful. Eight years ago, Marie McCook underwent the experimental procedure.
"I really don't think I'd be here without the transplant," McCook says.
Believers in the transplant therapy were quick to note that the other four studies are not yet completed and thus the findings could change -- they may yet show a longer-term benefit, or point to subgroups of women who do better with aggressive treatment. Also, they noted that the positive South African results could be because those doctors used different chemotherapy drugs and thus should not be disregarded.
Researcher Charles Hesdorffer complains the studies haven't run long enough to really answer the question.
"I don't think doctors are going to know what to do," says Dr. Charles Hesdorffer of Columbia-Presbyterian Medical Center. "I think patients are going to be totally confused as to what to do and there's going to be an incredible fight as to what the data means at this point."
Doctors had hoped that because of the increased risk and expense associated with high dose chemotherapy and stem cell transplant, it would havshown clear-cut benefits. So from that standpoint, this new research is disappointing, but by no means, is it the final word.
"What people would have liked I think is a yes or no," says Dr. John Durant of the American Society of Clinical Oncologists. "And we don't have very many yes or no's in medicine, and this is certainly not one of them."
Thursday's studies of the therapy have flaws: They started 10 years ago, and both standard chemotherapy and bone marrow transplants have improved since then. Too few patients entered the studies, making it hard to spot small reactions.
Cancer specialists fears that insurance companies will not want to continue to cover the $50,000-to-$150,000 procedure if final research results show it is less effective than believed.
All sides, including health insurers, are supporting a call for more research. But finding those clear answers will take years. For now, women and doctors are left with only a best guess on a life-and-death decision.