Cancer Drug Research

Despite advances in treating cancer, some forms of the disease remain resistant to all therapies and are often fatal. On the basis of findings suggesting that hydrazine sulfate may improve survival for some patients with advanced cancers, the National Cancer Institute sponsored three studies of the drug. All three of the studies, however, failed to show any benefit from hydrazine sulfate. The developer of hydrazine therapy alleged that the National Cancer Institute compromised its studies by allowing study patients to take tranquilizers, barbiturates, and alcohol, which he asserts are incompatible with hydrazine sulfate. GAO confirmed that all three trials allowed the use of tranquilizers to varying degrees and one trial allowed the use of barbiturates and alcohol. Retrospective analyses, however, found no evidence that the use of allegedly incompatible agents adversely affected the clinical trail results. Although GAO's work did not support the allegation that the studies were flawed, the National Cancer Institute should have ensured that complete and accurate records were kept on concurrent medications and possible alcohol use. Furthermore, National Cancer Institute investigators did not analyze this issue until recently, and the published results did not accurately describe the use of tranquilizers during one of these clinical trials.

GAO found that: (1) the three large NCI-sponsored clinical trials showed that the drug did not prolong cancer patients' survival; (2) controversy surrounding the trials focused on trial participants' use of tranquilizers, barbiturates, and alcohol, which were allegedly incompatible with the drug; (3) clinical trial records showed that participants used tranquilizers under varying circumstances, particularly for relief from vomiting; (4) the investigators believed that it was unethical to withhold antiemetic medications from patients undergoing chemotherapy; (5) subsequent analyses of patients' use of concurrent medications did not invalidate NCI conclusions that the drug was ineffective; (6) the Food and Drug Administration (FDA) may have contributed to the confusion surrounding the trials due to its more conservative position on how the drug should be administered to some patients; (7) although FDA approved more than 70 applications permitting the use of hydrazine sulfate, it cautioned physicians about their patients' use of tranquilizing agents while on the drug; (8) there were lapses in recordkeeping and reporting because NCI did not require complete and accurate research records on the patients' use of tranquilizing agents during the trials; and (9) NCI-sponsored investigators only recently analyzed this issue, since published results did not accurately describe the widespread use of tranquilizers during the trials.