Clinical and Translational Sciences: At the Intersection of Molecular and Individualized Medicine

Abstract

Advances in patient care exploit opportunities offered by discovery paradigms and their translation to therapeutic interventions. 1 Critical challenges in therapy at present include disease specificity, interpatient variability, and off‐target effects. 2 Molecular medicine provides a powerful catalyst for diagnostic and therapeutic platforms tailored to the genetic and molecular profile of individual patients to improve specificity, reduce variability, and minimize adverse events. 3 , 4 , 5 , 6 Indeed, advances in molecular medicine have transformed the contemporary therapeutic landscape from drug discovery through identification of drugable targets, development through stratification of patients and diseases, regulation through identifying pathways mediating off‐target effects, and utilization through matching patients with their optimal drug regimens. 2 , 7 , 8 This revolution in molecular therapeutics has entrained an evolution in biology and medicine. Insights achieved in molecular and genetic mechanisms directing cell, tissue, and organ function and their interface with the environment are translated to identify pathophysiological risks, define exogenous and endogenous mechanisms mediating disease susceptibility, target mechanism‐based therapeutic interventions, and tailor prevention and control strategies to provide integrated patient‐specific life‐long disease management. 9 , 10 The emerging field of clinical and translational science drives the leading edge advancing discovery from the laboratory bench to evidence‐based practice at the bedside, and beyond to populations, to transform the clinical enterprise and create predictive, personalized, and preemptive paradigms for customized patient‐specific therapeutic strategies. 3 , 5 , 9 , 11