What we are studying
The study drug was originally granted “accelerated approval” by the U.S. Food and Drug Administration (FDA) in 2000 to treat AML patients over age 60 who were experiencing their first leukemia relapse and who were not considered candidates for any other type of chemotherapy. This approval was granted was based on combined evidence from three clinical research studies that showed a 30% response rate among patients. Drugs approved under the “accelerated approval” process are required to submit additional data to confirm that the drug is indeed beneficial to patients. The required study that was conducted to validate the study drug's effectiveness was stopped early after it failed to show improved efficacy for patients treated with the study drug in addition to other chemotherapy medicines. As a result, the manufacturer and the FDA decided to voluntarily withdraw the study drug from commercial sale.
Although this study did not confirm that the study drug was effective when used in combination with other chemotherapy drugs, the study drug would be effective when used alone (i.e., not in combination with other chemotherapy drugs) to treat AML in a small subset (20-30%) of patients. You are being offered the study drug to treat your AML because you have failed other options.