Exceptional Responders Inspire Change: Lessons for Drug Development From the Bedside to the Bench and Back

Abstract

Targeted therapies have changed the landscape of cancer treatment, although they fail too many patients with advanced cancer. Still, insight gained from an exceptional responder has the power to identify new biomarkers of sensitivity that can unlock subsets of patients across anatomic disease sites who may also derive benefit and facilitate development of novel therapeutic strategies that may overcome resistance. The use of large-scale genomic profiling is a promising first step.