The Role of Precision Medicine in Neuroblastoma: Targeted Therapies and Personalized Approaches-A Narrative Review.

BACKGROUND AND AIMS: Neuroblastoma is the most common extracranial solid tumor and contributes the most to pediatric oncology deaths worldwide. Its clinical manifestations are heterogeneous, including relapses, and the prognosis remains poor, especially for high-risk patients. The principal interventions-surgery, chemotherapy, and radiotherapy-exacerbate the prognosis due to high morbidity and poor long-term remission rates. This study attempts to integrate available information on the genetic and molecular characteristics of neuroblastoma, focusing on how the potential of precision medicine is improving neuroblastoma diagnosis and treatment.

METHODS

This review aims to compile recent literature on the genomic, transcriptomic and epigenetic studies of neuroblastoma and its treatment. The review included the most recent molecular markers for the relevant pathway, the therapeutic targets that arise from them and precision medicine approaches that incorporate AI, CRISPR, and neoantigen vaccines at various stages of clinical trials.

RESULTS

Targeted medicines such MYCN amplification, ALK mutations, and chromosomal deletions including 1p36 and 11q have revealed important genetic drivers of neuroblastoma. Early-phase studies have shown encouraging reductions in tumor progression with MYCN modulators, ALK inhibitors and immunotherapies (anti-GD2 antibodies, B7-H3 CAR-T cells). Integrating omics-based markers and AI-directed therapy prediction more precisely classifies risk and customizes therapy.

CONCLUSION

Precision medicine has transformed neuroblastoma treatment by improving diagnostic specificity and therapeutic effectiveness while reducing toxicity. Among emerging strategies, ALK-targeted therapies and anti-GD2 immunotherapies show strong clinical potential, although further validation is still required to improve survival and quality of life in affected children.