New Approaches Towards Targeted Therapy for Childhood Neuroblastoma

Anticancer Res. 2023 Sep;43(9):3829-3839. doi: 10.21873/anticanres.16570.


Neuroblastoma (NB), comprising around 10% of all childhood neoplasms and 15% of pediatric cancer deaths is a heterogenous disease and can be divided into very low-, low-, intermediate- and high-risk NB. Treatment of very low/low-risk NB is usually based on observation, or surgery alone, whereas intermediate-risk NB is in addition to surgery treated with mild chemotherapy and roughly 80-95% of the patients are cured. In contrast, high-risk NB patients receive multimodal therapy with e.g. induction, consolidation, and maintenance therapy, which can include induction chemotherapy and surgery and in severe cases adding intensive chemotherapy and even autologous stem cell transplantation and radiotherapy. Unfortunately, however this treatment does not cure all patients and around 50% succumb to disease. For this purpose, new treatment options are urgently needed. In this review, we describe the complex molecular heterogeneity of NB, and potential new options for targeted therapy.

Keywords: ALK; MYCN; Neuroblastoma; immunotherapy; review; targeted therapy.

Publication types

  • Review

MeSH terms

  • Child
  • Combined Modality Therapy
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Induction Chemotherapy
  • Neuroblastoma* / drug therapy
  • Transplantation, Autologous