Perhaps the most striking example in any cancer of the benefit of molecularly targeted therapy is all-trans retinoic acid for acute promyelocytic leukemia. The introduction of this agent, and systematic study of how to use it, has increased the cure rate of APL from a maximum of 40 percent to over 70 percent in just 10 years. More recently, targeted therapy for chronic myelogenous leukemia with an Abl-specific tyrosine kinase inhibitor, STI571, has shown significant activity, including elimination of the causative genetic defect, in patients with advanced disease. As the application of chemotherapy in the hematologic malignancies led the way to improved chemotherapy for all cancers, so the development of these molecularly targeted therapies will serve as an important model for curing all cancers. Thus, a major expansion in translational research in the hematologic malignancies will provide a benefit for relieving the burden of cancer that far exceeds the frequency of these diseases.

(From Report of the Leukemia, Lymphoma, and Myeloma Progress Review Group)