Current and future strategies for relapsed neuroblastoma: challenges on the road to precision therapy

Area of Science:

• Pediatric Oncology
• Cancer Therapeutics
• Immunotherapy

Background:

• High-risk neuroblastoma (NB) has a poor prognosis, with over 50% of patients relapsing.
• Relapsed or refractory NB presents significant management challenges due to disease heterogeneity and treatment resistance.

Purpose of the Study:

• To review current treatment options for relapsed and refractory neuroblastoma.
• To discuss novel and emerging therapies on the horizon for neuroblastoma.

Main Methods:

• Review of current cytotoxic chemotherapy regimens including topotecan, irinotecan, cyclophosphamide, and temozolomide.
• Evaluation of I-meta-iodobenzylguanidine (MIBG) therapy for MIBG-avid relapsed disease.
• Discussion of immunotherapy, including anti-GD2 antibodies, and future cell-based approaches.
• Exploration of novel molecularly targeted agents in development for neuroblastoma.

Main Results:

• Current chemotherapy offers objective responses but limited long-term survival in relapsed/refractory NB.
• MIBG therapy shows objective responses in approximately one-third of MIBG-avid relapsed cases.
• The role of anti-GD2 immunotherapy in the relapse setting, particularly for bulky disease, requires further investigation.

Conclusions:

• Novel therapies, including targeted agents and cell-based immunotherapies, hold promise for overcoming limitations in current neuroblastoma treatment.
• Personalized approaches utilizing predictive biomarkers are crucial for effectively incorporating new agents into neuroblastoma treatment strategies.
• Future research should focus on combining novel therapies with conventional chemotherapy to improve outcomes for patients with relapsed or refractory neuroblastoma.