Searching for a cure for neuroblastoma: This review highlights the urgent need to find a cure for high-risk neuroblastoma. It provides an overview of the current treatment available and the drug discovery approach of seven drugs that reached clinical trial. Since 2017 there has been an acceleration in multi-target drug development, and the review concludes by arguing for a similar approach to be adopted in finding a cure for NB.

Abstract

Neuroblastoma (NB) is a childhood malignant tumour belonging to a group of embryonic tumours originating from progenitor cells of the sympathoadrenal lineage. The heterogeneity of NB is reflected in the survival rates of those with low and intermediate risk diseases who have survival rates ranging from 85 to 90 %. However, for those identified with high-risk Stage 4 NB, the treatment options are much more limited. For this group, current treatment consists of immunotherapy (monoclonal antibodies) in combination with anti-cancer drugs and has a 40 to 50 % survival rate. The purpose of this review is to summarise NB research from a medicinal chemistry perspective and to highlight advances in targeted drug therapy in the field. The review examines the medicinal chemistry of a number of drugs tested in research, some of which are currently under clinical trial. It concludes by proposing that future medicinal chemistry research into NB should consider other possible target therapies and adopt a multi-target drug approach rather than a one-drug-one-target approach for improved efficacy and less drug-drug interaction for the treatment of NB Stage 4 (NBS4) patients.