ST1571: A paradigm of new agents for cancer therapeutics

ST1571 exemplifies the successful development of a rationally designed, molecularly targeted therapy for the treatment of a specific cancer. This article reviews the identification of Bcr-Abl as a therapeutic target in chronic myelogenous leukemia and the steps in the development of an agent to specifically inactivate this abnormality. Issues related to clinical trials of molecularly targeted agents are discussed, including dose selection, optimizing therapy, and predicting response, as are possible mechanisms of resistance to ST1571. Lastly, the potential use of ST1571 in other malignancies and the translation of this paradigm to other malignancies are explored. (C) 2001 by American Society of Clinical Oncology.