ABSTRACT

In the past quarter century, tremendous strides have been made in our understanding of the molecular mechanisms involved in human disease. These advances have led to a dramatic change in the drug discovery paradigm within the pharmaceutical industry, which has evolved from a random screening approach to a target-driven drug discovery process. The progress made within the field of chronic myeloid leukemia (CML) is emblematic of this new paradigm, and serves as an example of how basic research is setting the stage to bring therapeutics to the bedside. As described in other chapters in this volume, many molecular entities have been identified as potential targets for therapeutic intervention. I will limit my discussion to the molecular targets and signaling pathways shown in Figure 32.1, highlighting strategies and technological advances that show promise for developing pharmaceuticals targeting BCR/ABL function and that can be applied to other molecular entities not specifically mentioned.