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Conducting Clinical Trials

  • Posted: 04/26/2001
  • Reviewed: 04/12/2005

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High-Dose Chemotherapy for Breast Cancer: History

Over the past 20 years, more than 15,000 women with breast cancer have been treated with an arduous yet largely unproven procedure: high doses of chemotherapy followed by blood cell transplants to replenish the bone marrow damaged by the chemotherapy.

In the early 1990s, after a few encouraging preliminary reports, breast cancer patients and advocates began demanding the treatment. Some state legislatures responded by mandating that insurance companies pay for the intensive procedure, which can cost up to $100,000 per case, much more than conventional treatments. By mid-decade, more people were receiving the treatment for breast cancer than for any other cancer.

However, the vast majority of these women who received the high-dose regimens did so outside of a clinical trial, which meant that the success or failure of the treatment could not be reliably established. Results from well-conducted, carefully monitored, randomized trials were urgently needed to figure out which breast cancer patients, if any, might benefit from high-dose therapy plus transplants, said Jeff Abrams, M.D., a breast cancer researcher at NCI.

So in the late 1990s, multiple randomized studies were begun. In early 2000, reports from three of these clinical trials were published. Two showed that high doses of chemotherapy were no more effective than standard chemotherapy for women with advanced or high-risk breast cancer. Results from the third study contradicted these findings and came out in favor of the high-dose treatment. However, this study was later discounted after the lead South African researcher admitted fraud and misconduct.

These developments left the practice of high-dose chemotherapy for breast cancer in limbo. At that point, the American Society of Clinical Oncology (ASCO) recommended that women receive the treatment only if they were taking part in a "high-quality" clinical trial. Editorials in the March 18, 2000, issue of the Lancet and the April 13, 2000, New England Journal of Medicine made the same argument.

In another sign of support for the clinical trials approach, in early 2000 one of the nation's largest insurers, Aetna/U.S. Healthcare, announced that it will pay for high-dose chemotherapy plus transplants only for patients enrolled in federally sponsored clinical trials. The company previously reimbursed expenses for women who were not in clinical trials.

Researchers at the National Cancer Institute (NCI) have long supported this approach. As of spring 2001, four large national trials sponsored by NCI had all completed enrollment, with results beginning to become available.