Changing Paradigms in Cancer Clinical Trials

Abstract

The advances made in cancer biology and the genetics of cancer have rapidly provided us with a better fundamental understanding of cancer; but progress in translating such knowledge into medical practice is relatively slow. Indeed, important examples of translational approaches may already be found in the areas of drug discovery and development, disease diagnosis and classification, and the selection of therapeutic regimens that are specific to the individual. These new developments require novel approaches to incorporate the quantity and diversity of information for evaluating therapies and potential diagnostic and prognostic markers. For the translation of knowledge into practice to be effective requires the conduct of studies with appropriate design and analyses based on sound statistical principles. Research is under way to find new ways of exploring the use of biomarkers in cancer detection and treatment that will in turn require a new generation of clinical trials that modernize the processes and methods used to examine the safety and efficacy of novel, gene-based therapies without sacrificing high standards. This chapter revisits some of the basic components of clinical trial design within the context of timely areas of vaccine trials, cancer stem cell trials, and trials of epigenetic targeted therapies. We highlight the molecular and cancer biology influences on trial design in each area to illustrate the outstanding need for new and improved cancer clinical trial design and analyses.