Related Experiment Video
Updated: May 11, 2026
Nanoparticle Delivery of an Oligonucleotide Payload in a Glioblastoma Multiforme Animal Model
Published on: September 27, 2024
Emerging drugs for neuroblastoma
Victoria Castel1, Vanessa Segura, Pablo Berlanga
1Unidad de Oncología Pediátrica, Hospital Universitario y Politécnico La Fe, Torre G, 2° Planta, Bulevar Sur s/n, 46026 Valencia, Spain. castel_vic@gva.es
Introduction:
Neuroblastoma accounts for 8 - 10% of pediatric cancers and is responsible for 15% of childhood cancer deaths. Despite multimodality treatment, the overall survival (OS) and event-free survival (EFS) in high-risk patients remain suboptimal. More than half of children diagnosed with high-risk neuroblastoma either do not respond to conventional therapies or relapse after treatment.
Areas Covered:
This review discusses about the unmet medical needs for new therapeutic options against high-risk neuroblastoma. New drugs and therapeutic strategies that are under development in clinical trials, which are currently recruiting patients.
Expert Opinion:
There is a need to improve the response rate of induction chemotherapy, which is not effective in a third of patients and also the other components of the current treatment, little efficacious in avoiding the relapses. Few drugs have been introduced as upfront therapy in the last years. Topotecan, irinotecan and temozolomide are expected to improve the response in high-risk neuroblastoma, but their impact on OS and EFS is unknown. Anti-GD2 antibodies combined with other immunomodulators (IL-2, GM-CSF) are an important advance in the treatment of these children. Nevertheless, the hope is put in the new drugs directed to molecular targets of neuroblastoma. Anti-angiogenic drugs, ALK antagonist and PI3K/Akt/mTOR inhibitors are among the most promising.
Insights
High-risk neuroblastoma treatment remains challenging, with suboptimal survival rates. Emerging therapies, including targeted drugs and immunotherapies, offer new hope for improving outcomes in pediatric cancer patients.
Area of Science:
- Pediatric Oncology
- Cancer Therapeutics
- Clinical Trials
Background:
- Neuroblastoma is a significant cause of pediatric cancer mortality (15%) and diagnosis (8-10%).
- High-risk neuroblastoma patients exhibit poor overall survival (OS) and event-free survival (EFS) despite current treatments.
- Over 50% of high-risk neuroblastoma cases show resistance or relapse post-therapy.
Purpose of the Study:
- To review unmet medical needs in high-risk neuroblastoma treatment.
- To highlight novel therapeutic options currently in clinical trials for pediatric neuroblastoma.
Main Methods:
- Review of current literature on neuroblastoma treatment strategies.
- Analysis of ongoing clinical trials for new neuroblastoma drugs and therapies.
Main Results:
- Existing chemotherapy regimens have limited efficacy in a third of patients and fail to prevent relapses.
- Newer agents like topotecan, irinotecan, and temozolomide show potential but require further OS/EFS data.
- Anti-GD2 antibodies with immunomodulators represent a significant advancement.
Conclusions:
- Improved induction chemotherapy response rates and reduced relapse rates are critical.
- Targeted therapies, including anti-angiogenic drugs, ALK antagonists, and PI3K/Akt/mTOR inhibitors, are promising for high-risk neuroblastoma.
