Transforming Biomarker Development with Exceptional Responders.

Curative therapy for cancer patients with advanced-stage disease remains elusive. While rare outlier responses to anticancer therapies exist, barriers limit our understanding of the molecular and genetic basis of such profound, life-altering responses. Here, we describe how phenotype-to-genotype studies are elucidating the molecular underpinnings of outlier responses and informing strategies to extend such unprecedented sensitivity to broader molecularly defined patient populations.