Established and Emerging Therapies for High-Risk Neuroblastoma

Area of Science:

• Pediatric Oncology
• Cancer Immunology
• Translational Cancer Research

Background:

• High-risk neuroblastoma (HRNB) remains a leading cause of pediatric cancer mortality despite intensive multimodal treatments.
• Current therapeutic outcomes for HRNB have plateaued, with refractory and relapsed cases exhibiting poor prognoses.
• Intensive treatments for HRNB cause significant toxicities, impacting patients' quality of life and long-term health.

Purpose of the Study:

• To review the current standard of care for high-risk neuroblastoma.
• To focus on emerging immunotherapies, including cellular therapy, for HRNB treatment.
• To identify novel approaches for improving survival rates and reducing late effects in pediatric cancer.

Main Methods:

• Literature review of current and emerging HRNB treatment modalities.
• Analysis of advancements in chemotherapy, surgery, stem cell transplantation, radiation, and immunotherapy.
• Examination of novel targeted agents, radionucleotides, and cellular therapies.

Main Results:

• Frontline therapies for HRNB include chemotherapy, surgery, autologous stem cell transplantation (ASCT), radiation, and immunotherapy.
• Emerging strategies involve anti-GD2 immunotherapy, iodine-131 (131I)-metaiodobenzylguanidine (MIBG) therapy, and targeted agents.
• Innovative approaches for refractory/relapsed HRNB include novel targeted therapies, immunotherapy, and radionucleotides.

Conclusions:

• Novel therapeutic strategies are crucial to overcome the plateau in HRNB outcomes.
• Emerging immunotherapies and cellular therapies show promise for improving survival and reducing treatment-related toxicities.
• Integrating new modalities into frontline and salvage regimens is key to advancing pediatric cancer care.