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New therapeutic targets for the treatment of high-risk neuroblastoma
1Division of Pediatric Hematology/Oncology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.
Abstract:
High-risk neuroblastoma remains a major problem in pediatric oncology, accounting for 15% of childhood cancer deaths. Although incremental improvements in outcome have been achieved with the intensification of conventional chemotherapy agents and the addition of 13-cis-retinoic acid, only one-third of children with high-risk disease are expected to be long-term survivors when treated with current regimens. In addition, the cost of cure can be quite high, as surviving children remain at risk for additional health problems related to long-term toxicities of treatment. Further advances in therapy will require the targeting of tumor cells in a more selective and efficient way so that survival can be improved without substantially increasing toxicity. In this review we summarize ongoing clinical trials and highlight new developments in our understanding of the molecular biology of neuroblastoma, emphasizing potential targets or pathways that may be exploitable therapeutically.
Insights
High-risk neuroblastoma is a significant challenge in pediatric cancer, with current treatments offering limited long-term survival. New therapeutic strategies are needed to improve outcomes and reduce treatment toxicity in children.
Area of Science:
- Pediatric Oncology
- Molecular Biology
- Cancer Therapeutics
Background:
- High-risk neuroblastoma accounts for 15% of childhood cancer deaths.
- Current treatment regimens offer limited long-term survival rates (approximately one-third).
- Existing therapies can lead to significant long-term toxicities and health problems in survivors.
Purpose of the Study:
- To review ongoing clinical trials for high-risk neuroblastoma.
- To highlight advances in understanding neuroblastoma's molecular biology.
- To identify potential therapeutic targets and pathways for improved treatment.
Main Methods:
- Review of current clinical trials.
- Analysis of recent research on neuroblastoma molecular biology.
- Identification of novel therapeutic targets and pathways.
Main Results:
- Incremental improvements in outcomes with intensified chemotherapy and 13-cis-retinoic acid.
- Significant need for more selective and efficient tumor-targeting therapies.
- Emerging understanding of molecular pathways offers new therapeutic avenues.
Conclusions:
- Further advances require targeted approaches to improve survival without increasing toxicity.
- Exploiting molecular targets holds promise for more effective neuroblastoma treatment.
- Ongoing research and clinical trials are crucial for developing next-generation therapies.
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