A 2035 Clinical Research Vision and Roadmap for High-Risk Neuroblastoma

Abstract

Despite the introduction of anti-GD2 antibody therapy, outcomes for children with high-risk neuroblastoma remain poor, with low cure rates and a high proportion of survivors facing long-term sequelae. In this report, leaders from international cooperative groups and patient advocacy organizations review lessons learnt, identify current challenges, and provide a vision to bring new agents into frontline therapy to increase cure rates and reduce long-term toxicities over the next decade. The implementation of this vision requires improved global collaboration, incorporation of novel biomarkers, and a strengthened interaction with the regulatory landscape.