Molecular Annotation for Treatment of Childhood Cancers Trial: Neuroblastoma (MATCH-NB)
Neuroblastoma remains a devastating clinical problem and continues to be a leading cause of childhood cancer morbidity and mortality despite dramatic increases in therapy. Recent developments in cancer research are changing our understanding of neuroblastoma biology and treatment on the basis of the molecular underpinnings of individual tumors, and are challenging us to design more rational and less toxic therapies. We propose a paradigm-shifting collaborative clinical trial for children with relapsed or refractory neuroblastoma to identify the genetic features of the cancer at time of relapse that predict drug efficacy, and to provide combination therapies to target these vulnerabilities. We have designed a program to allow for the addition of new therapies based on ongoing laboratory studies, with the overarching goal of substantially changing how we think about neuroblastoma at relapse and how these insights may inform the design of upfront clinical trials for newly diagnosed patients with high-risk neuroblastoma.