TRANSLATIONAL RESEARCH - WALKING THE BRIDGE BETWEEN IDEA AND CURE - 17TH BRUCE-F.-CAIN-MEMORIAL-AWARD-LECTURE

Advances in the understanding of normal and malignant cell biology are allowing the development of biologically targeted drugs directed at s pecific differences between host and tumor. The array of potential new targets is vast, but drugs currently in development are targeted at c ell-cycle regulators, growth factors and their receptors, signal trans duction intermediates, angiogenesis, and the mechanisms that mediate a poptosis and DNA repair. Recent results raise the possibility that nov el biologically targeted agents, perhaps in combination with tradition al cytotoxic agents, may finally cure cancer. However, the development of a biologically targeted drug raises unique challenges in the desig n of clinical trials to demonstrate its efficacy, and despite the prom ising preclinical data that exist for most of the agents in developmen t, the clinical trial remains the critical, final step across the brid ge from basic research to clinical application. In this review, we dis cuss some of the challenges in the clinical development of biologicall y targeted agents and the implications for clinical trial design.