Initial Testing of the Investigational NEDD8 Activating Enzyme Inhibitor MLN4924 by the Pediatric Preclinical Testing Program

摘要

BackgroundMLN4924 is an investigational first-in-class small molecule inhibitor of NEDD8-activating enzyme (NAE). NAE is an essential component of the NEDD8 conjugation pathway, controlling the activity of a subset of ubiquitin-proteasome system (UPS) E3 ligases, multiprotein complexes that transfer ubiquitin molecules to substrate proteins.