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Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
Published on: August 25, 2023
Genetic discoveries and treatment advances in neuroblastoma
Rochelle Bagatell1, Susan L Cohn
1aDivision of Oncology, The Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania bDepartment of Pediatrics, Comer Children's Hospital, University of Chicago, Chicago, Illinois, USA.
Purpose Of Review:
Major advances in our understanding of the genetic basis of neuroblastoma, and the role somatic alterations play in driving tumor growth, have led to improvements in risk-stratified therapy and have provided the rationale for targeted therapies. In this review, we highlight current risk-based treatment approaches and discuss the opportunities and challenges of translating recent genomic discoveries into the clinic.
Recent Findings:
Significant progress in the treatment of neuroblastoma has been realized using risk-based treatment strategies. Outcome has improved for all patients, including those classified as high-risk, although survival remains poor for this cohort. Integration of whole-genome DNA copy number and comprehensive molecular profiles into neuroblastoma classification systems will allow more precise prognostication and refined treatment assignment. Promising treatments that include targeted systemic radiotherapy, pathway-targeted small molecules, and therapy targeted at cell surface molecules are being evaluated in clinical trials, and recent genomic discoveries in relapsed tumor samples have led to the identification of new actionable mutations.
Summary:
The integration of refined treatment stratification based on whole-genome profiles with therapeutics that target the molecular drivers of malignant behavior in neuroblastoma has the potential to dramatically improve survival, with decreased toxicity.
Insights
Advances in understanding neuroblastoma genetics improve risk-stratified therapy. Integrating genomic data with targeted treatments offers improved survival and reduced toxicity for patients.
Area of Science:
- Pediatric Oncology
- Cancer Genomics
- Neuroblastoma Research
Background:
- Neuroblastoma genetics and somatic alterations are increasingly understood.
- This knowledge has improved risk-stratified therapy and informed targeted treatment strategies.
Purpose of the Study:
- To review current risk-based treatment approaches for neuroblastoma.
- To discuss translating genomic discoveries into clinical practice for neuroblastoma.
Main Methods:
- Review of current literature on neuroblastoma genetics and treatment.
- Analysis of risk-based treatment strategies and genomic profiling integration.
Main Results:
- Risk-based strategies have improved neuroblastoma outcomes, including for high-risk patients.
- Whole-genome and molecular profiling enhance prognostication and treatment assignment.
- New targeted therapies and actionable mutations are identified in clinical trials and relapsed tumors.
Conclusions:
- Integrating genomic profiles for refined treatment stratification can significantly improve neuroblastoma survival.
- Targeting molecular drivers of neuroblastoma offers potential for improved outcomes with decreased toxicity.
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