Research Advances in Small Molecule Targeted Drugs for Pediatric Neuroblastoma

Malignant tumors have become the second leading cause of death in children, with neuroblastoma(NB) being one of the most common pediatric malignant solid tumors, accounting for 15% of childhood cancer-related mortality. The high heterogeneity exhibited by NB poses challenges to its treatment, which currently encompasses surgical resection, chemotherapy, stem cell transplantation, radiation therapy, anti-GD2 monoclonal antibody based immunotherapy, and the differentiating agent 13-cis retinoic acid therapy. So new therapeutic approaches are urgently needed to enhance the efficacy, reduce recurrence rates, and improve survival rates in high-risk NB children. Small-molecule targeted drugs have the advantages of strong selectivity, low toxicity and stable structure, and have been extensively employed in precision medicine for various tumors. With advancements in understanding the pathogenesis of NB, numerous molecular signatures have been identified as targets for therapeutic intervention. This review provides a comprehensive summary of current potential targets for NB along with ongoing research on small molecule targeted drugs, in order to facilitate further development of targeted therapies in pediatric NB.