title: At the frontier of progress for paediatric oncology: the neuroblastoma paradigm.
abstract: BACKGROUND
  Neuroblastoma is one of the commonest and deadliest forms of childhood cancer and major initiatives are ongoing to improve the outcome of these patients.
  
  
  METHODS
  Data for this review were obtained from PubMed and abstracts from the American Society of Clinical Oncology and Advances in Neuroblastoma Research.
  
  
  RESULTS
  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.
  
  
  RESULTS
  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.
  
  
  CONCLUSIONS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Paediatric Drug Development Team, Di visions of Cancer Therapeutics and Clinical Studies, The Institute of Cancer Research, Sutton SM2 5NG, UK.
authors.forename: Lucas
authors.initials: L
authors.lastname: Moreno
authors.forename: Lynley V
authors.initials: LV
authors.lastname: Marshall
authors.forename: Andrew D J
authors.initials: AD
authors.lastname: Pearson
mesh_terms: D035843:Biomedical Research; D002648:Child; D003299:Cooperative Behavior; D055808:Drug Discovery; D006801:Humans; D007391:International Cooperation; D008495:Medical Oncology; D009447:Neuroblastoma / Q000235:genetics / Q000628:therapy*; D010372:Pediatrics
keywords: biomarkers; childhood cancer; drug development; neuroblastoma; phase I; phase II