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Published on: July 29, 2011
Methodological advances in the discovery of novel neuroblastoma therapeutics
Miguel F Segura1, Aroa Soriano1, Josep Roma1
1Pediatric Oncology and Hematology Department, Translational Research in Child and Adolescent Cancer, Vall d'Hebron Institut de Recerca (VHIR), Barcelona. Universitat Autònoma de Barcelona, Bellaterra, Spain.
Introduction:
Neuroblastoma is a cancer of the sympathetic nervous system that causes up to 15% of cancer-related deaths among children. Among the ~1,000 newly diagnosed cases per year in Europe, more than half are classified as high-risk, with a 5-year survival rate <50%. Current multimodal treatments have improved survival among these patients, but relapsed and refractory tumors remain a major therapeutic challenge. A number of new methodologies are paving the way for the development of more effective and safer therapies to ultimately improve outcomes for high-risk patients.
Areas Covered:
The authors provide a critical review on methodological advances aimed at providing new therapeutic opportunities for neuroblastoma patients, including preclinical models of human disease, generation of omics data to discover new therapeutic targets, and artificial intelligence-based technologies to implement personalized treatments.
Expert Opinion:
While survival of childhood cancer has improved over the past decades, progress has been uneven. Still, survival is dismal for some cancers, including high-risk neuroblastoma. Embracing new technologies (e.g. molecular profiling of tumors, 3D in vitro models, etc.), international collaborative efforts and the incorporation of new therapies (e.g. RNA-based therapies, epigenetic therapies, immunotherapy) will ultimately lead to more effective and safer therapies for these subgroups of neuroblastoma patients.
Insights
New methodologies offer hope for high-risk neuroblastoma, a childhood cancer with poor survival rates. Advances in preclinical models, omics data, and AI aim to create safer, more effective personalized treatments.
Area of Science:
- Pediatric Oncology
- Cancer Research
- Translational Medicine
Background:
- Neuroblastoma is a sympathetic nervous system cancer causing significant childhood cancer deaths.
- High-risk neuroblastoma has a dismal 5-year survival rate (<50%) despite multimodal treatments.
- Relapsed and refractory tumors present a major therapeutic challenge in neuroblastoma.
Purpose of the Study:
- To critically review methodological advances for novel neuroblastoma therapies.
- To highlight new therapeutic opportunities for neuroblastoma patients.
- To discuss strategies for improving outcomes in high-risk neuroblastoma.
Main Methods:
- Review of preclinical models of human neuroblastoma.
- Analysis of omics data for therapeutic target discovery.
- Exploration of artificial intelligence (AI)-based technologies for personalized treatment.
Main Results:
- Methodological advances are paving the way for improved neuroblastoma therapies.
- New technologies like molecular profiling and 3D in vitro models are crucial.
- Emerging therapies include RNA-based, epigenetic, and immunotherapies.
Conclusions:
- International collaboration and embracing new technologies are vital for high-risk neuroblastoma.
- Personalized treatments hold promise for improving survival rates.
- Development of safer and more effective therapies is essential for neuroblastoma patients.
