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Updated: Aug 14, 2026
Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
[Targeted therapies in pediatric oncology: a new therapeutic approach?]
C Jubert1, B Geoerger, J Grill
1UPRES EA 3535, Département de Pédiatrie, Pharmacologie et Nouveaux Traitements des Cancers, Institut Gustave-Roussy, 39, rue Camille-Desmoulins, 94805 Villejuif cedex, France.
Abstract:
A multidisciplinary therapeutic approach has led to significant increase in survival of children with cancer, however often with a high rate of severe sequela. Better understanding in tumor cell biology and transformation process allowed to describe active tyrosine kinases (mainly growth factor receptors) as a new target for cancer treatment. This review presents 2 approaches to target receptor tyrosine kinase activity: on one hand, antibodies that target the extracellular domain, the natural ligand binding site, and on the other hand, small inhibiting molecules, such as imatinib, targeted against the activated intracellular receptor tyrosine kinase. We focus on their clinical development and current application in the treatment of childhood cancer. Targeted therapies are in full rise and new perspectives are explored, such as their association to other treatment modalities and the targeting of microenvironment. This new therapeutic approach necessitates well designed clinical trials that include relevant biomarkers to evaluate its real therapeutic potential.
Insights
Targeted therapies, like tyrosine kinase inhibitors, improve childhood cancer survival but can cause side effects. New approaches focus on targeting tumor biology and the microenvironment for better outcomes.
Area of Science:
- Oncology
- Molecular Biology
- Pharmacology
Background:
- Multidisciplinary cancer treatment improves survival in children but often results in severe sequelae.
- Advances in understanding tumor cell biology have identified active tyrosine kinases as novel therapeutic targets.
Purpose of the Study:
- To review current approaches targeting receptor tyrosine kinase activity in pediatric cancers.
- To discuss the clinical development and application of targeted therapies.
Main Methods:
- Review of literature on antibodies targeting extracellular domains of receptor tyrosine kinases.
- Review of literature on small molecule inhibitors (e.g., imatinib) targeting intracellular receptor tyrosine kinases.
Main Results:
- Two main strategies exist: extracellular domain-targeting antibodies and intracellularly-acting small molecule inhibitors.
- These targeted therapies are increasingly used in treating childhood cancers.
Conclusions:
- Targeted therapies represent a significant advancement in pediatric oncology.
- Future directions include combination therapies, targeting the tumor microenvironment, and the need for well-designed trials with biomarkers.
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