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Molecular profiling of childhood cancer: Biomarkers and novel therapies
Federica Saletta1, Carol Wadham2, David S Ziegler3
1Children's Cancer Research Unit, Kids Research Institute, Westmead 2145, New South Wales, Australia.
Background:
Technological advances including high-throughput sequencing have identified numerous tumor-specific genetic changes in pediatric and adolescent cancers that can be exploited as targets for novel therapies.
Scope Of Review:
This review provides a detailed overview of recent advances in the application of target-specific therapies for childhood cancers, either as single agents or in combination with other therapies. The review summarizes preclinical evidence on which clinical trials are based, early phase clinical trial results, and the incorporation of predictive biomarkers into clinical practice, according to cancer type.
Major Conclusions:
There is growing evidence that molecularly targeted therapies can valuably add to the arsenal available for treating childhood cancers, particularly when used in combination with other therapies. Nonetheless the introduction of molecularly targeted agents into practice remains challenging, due to the use of unselected populations in some clinical trials, inadequate methods to evaluate efficacy, and the need for improved preclinical models to both evaluate dosing and safety of combination therapies.
General Significance:
The increasing recognition of the heterogeneity of molecular causes of cancer favors the continued development of molecularly targeted agents, and their transfer to pediatric and adolescent populations.
Insights
Target-specific therapies show promise for treating pediatric and adolescent cancers, especially when combined with other treatments. Further research is needed to overcome challenges in clinical application and improve preclinical models for these novel agents.
Area of Science:
- Oncology
- Genetics
- Pharmacology
Background:
- High-throughput sequencing reveals tumor-specific genetic alterations in pediatric and adolescent cancers.
- These genetic changes present opportunities for developing novel targeted therapies.
Purpose of the Study:
- To review recent advances in targeted therapies for childhood cancers.
- To summarize preclinical data, early clinical trial results, and biomarker integration.
- To discuss challenges and future directions for targeted therapy implementation.
Main Methods:
- Comprehensive review of preclinical studies and early-phase clinical trials.
- Analysis of the incorporation of predictive biomarkers in clinical practice.
- Focus on target-specific agents as single agents or in combination therapies.
Main Results:
- Molecularly targeted therapies can significantly enhance childhood cancer treatment, particularly in combination regimens.
- Evidence supports the value of targeted agents in pediatric and adolescent oncology.
- Challenges include unselected patient populations, efficacy evaluation methods, and preclinical model limitations.
Conclusions:
- Targeted therapies offer a valuable addition to the pediatric cancer treatment armamentarium.
- Continued development and transfer of molecularly targeted agents are favored by cancer heterogeneity.
- Overcoming clinical implementation challenges is crucial for realizing the full potential of these therapies.
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