Neuroblastoma: evolving therapies for a disease with many faces

Neuroblastoma is the most common extra-cranial solid tumor in children and has a heterogeneous clinical presentation and course. Clinical and biologic features of this disease have been used to develop risk-based therapy. Patients with low-risk disease can be treated with surgery alone. Patients with intermediate-risk features have an excellent prognosis after treatment with surgery and a relatively short course of standard dose chemotherapy. Unfortunately, most children with neuroblastoma present with advanced disease. More than 60% of patients with high-risk features will succumb to their disease despite intensive therapy including a myeloablative consolidation. Research efforts to understand the biologic basis of neuroblastoma and to identify new, more effective therapies are essential to improve the outcome for these children.