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Proton Therapy Delivery and Its Clinical Application in Select Solid Tumor Malignancies
Published on: February 6, 2019
Targeted treatment of solid tumors in pediatric precision oncology
Ilaria Bertacca1,2, Francesco Pegoraro1,2, Annalisa Tondo1
1Paediatric Hematology/Oncology Department, Meyer Children's Hospital, Firenze, Italy.
Abstract:
The treatment of childhood solid cancer has markedly evolved in recent years following a refined molecular characterization and the introduction of novel targeted drugs. On one hand, larger sequencing studies have revealed a spectrum of mutations in pediatric tumors different from adults. On the other hand, specific mutations or immune dysregulated pathways have been targeted in preclinical and clinical studies, with heterogeneous results. Of note, the development of national platforms for tumor molecular profiling and, in less measure, for targeted treatment, has been essential in the process. However, many of the available molecules have been tested only in relapsed or refractory patients, and have proven poorly effective, at least in monotherapy. Our future approaches should certainly aim at improving the access to molecular characterization, to obtain a deeper picture of the distinctive phenotype of childhood cancer. In parallel, the implementation of access to novel drugs should not only be limited to basket or umbrella studies but also to larger, multi-drug international studies. In this paper we reviewed the molecular features and the main available therapeutic options in pediatric solid cancer, focusing on available targeted drugs and ongoing investigations, aiming at providing a useful tool to navigate the heterogeneity of this promising but complex field.
Insights
Pediatric solid cancer treatment advances with molecular profiling and targeted drugs. Future research needs broader access to characterization and international drug trials for better outcomes in childhood cancers.
Area of Science:
- Oncology
- Pediatric Medicine
- Molecular Biology
Background:
- Childhood solid cancer treatment is evolving due to molecular insights and targeted therapies.
- Pediatric tumors exhibit distinct mutations compared to adult cancers.
- National platforms for molecular profiling are crucial for advancing pediatric cancer care.
Purpose of the Study:
- To review molecular features of pediatric solid cancers.
- To discuss current targeted drug options and ongoing investigations.
- To provide a guide for navigating targeted therapies in pediatric oncology.
Main Methods:
- Review of recent molecular characterization studies in pediatric tumors.
- Analysis of preclinical and clinical data for targeted drugs in childhood cancers.
- Examination of national platforms for tumor molecular profiling and targeted treatment.
Main Results:
- Significant molecular differences exist between adult and pediatric tumors.
- Targeted therapies show heterogeneous results, particularly in relapsed/refractory cases.
- Current targeted drugs often have limited efficacy as monotherapy.
Conclusions:
- Improved access to molecular characterization is essential for understanding childhood cancer phenotypes.
- Future strategies require broader access to novel drugs through international, multi-drug studies.
- Navigating the complexity of pediatric solid cancer requires a comprehensive understanding of molecular features and therapeutic options.
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