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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
Expert Opinion on Emerging Drugs
|May 23, 2013
Summary
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.
