Neuroblastoma

Neuroblastoma, a malignant neoplasm of the sympathetic nervous system, is the most common extracranial solid tumour in childhood. Since its first description in the nineteenth century, its highly heterogeneous clinical presentation has challenged clinicians and fascinated basic researchers. Neuroblastoma serves as a paradigm for the prognostic utility of biological and clinical data and the potential to tailor therapy for patient cohorts at low, intermediate, and high risk for recurrence. This chapter presents an overview of the key genetic, molecular, histological, and clinical features of neuroblastoma, as well as current risk-stratification strategies and therapeutic approaches. It also highlights how our understanding of tumour pathogenesis, coupled with molecular analyses, has illuminated critical signal transduction pathways and key molecules involved in neuroblastoma tumourigenesis, pointing to novel therapeutic targets for clinical development. Future treatment avenues for relapsed neuroblastoma are discussed, including new drugs targeting ALK, MYC/MYCN, histone deacetylases, or MDM2/TP53.