At the frontier of progress for paediatric oncology: the neuroblastoma paradigm

Abstract

Neuroblastoma is one of the commonest and deadliest forms of childhood cancer and major initiatives are ongoing to improve the outcome of these patients. Data for this review were obtained from PubMed and abstracts from the American Society of Clinical Oncology and Advances in Neuroblastoma Research. Collaborative clinical trials have led to major improvements in treatment outcomes for low and intermediate risk neuroblastoma, and international initiatives such as the International Neuroblastoma Risk Group have produced a very refined risk stratification incorporating clinical and biological risk factors. Despite many efforts, the outcome for high-risk neuroblastoma is still poor and the only new strategy incorporated into frontline treatment is anti-GD2 immunotherapy. It is unclear how new drugs targeting specific molecular aberrations will be incorporated. Genomic characterization and drug development have undergone major advances in the last 5 years leading to a much deeper understanding of tumour biology as well as active biomarker-driven preclinical and clinical research on new molecules that will hopefully progress faster and more efficiently into frontline combination treatment strategies. Significant effort remains to be done in integrating the different new strategies, combining new molecularly targeted agents to maximize therapeutic benefit and incorporate immunotherapy together with targeted therapies.