Development of STI571 and its use in chronic myelogenous leukemia and other malignancies

Chronic myelogenous leukemia (CML) is a clonal hematopoetic stem cell disorder characterized by the (9:22) translocation and resultant production of the constitutively activated Bcr-Abl tyrosine kinase. Characterized clinically by marked myeloid proliferation, it terminates invariably in an acute leukemia. Conventional therapeutic options include interferon-based regimens and stem cell transplantation, with stem cell transplantation being the only curative therapy. Through rational drug development, STI571, a Bcr-Abl tyrosine kinase inhibitor, has emerged as a paradigm for gene product targeted therapy, offering new hope for expanded treatment options for patients with CML.