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Updated: Oct 4, 2025
Oncogenic Gene Fusion Detection Using Anchored Multiplex Polymerase Chain Reaction Followed by Next Generation Sequencing
Published on: July 5, 2019
Molecular Targets for Novel Therapeutics in Pediatric Fusion-Positive Non-CNS Solid Tumors
Wen-I Chang1,2,3, Claire Lin1, Nicholas Liguori1
1Laboratory of Translational Oncology and Experimental Cancer Therapeutics, The Warren Alpert Medical School, Brown University, Providence, RI, United States.
Abstract:
Chromosomal fusions encoding novel molecular drivers have been identified in several solid tumors, and in recent years the identification of such pathogenetic events in tumor specimens has become clinically actionable. Pediatric sarcomas and other rare tumors that occur in children as well as adults are a group of heterogeneous tumors often with driver gene fusions for which some therapeutics have already been developed and approved, and others where there is opportunity for progress and innovation to impact on patient outcomes. We review the chromosomal rearrangements that represent oncogenic events in pediatric solid tumors outside of the central nervous system (CNS), such as Ewing Sarcoma, Rhabdomyosarcoma, Fibrolamellar Hepatocellular Carcinoma, and Renal Cell Carcinoma, among others. Various therapeutics such as CDK4/6, FGFR, ALK, VEGF, EGFR, PDGFR, NTRK, PARP, mTOR, BRAF, IGF1R, HDAC inhibitors are being explored among other novel therapeutic strategies such as ONC201/TIC10.
Insights
Chromosomal fusions are key drivers in rare pediatric solid tumors. Identifying these genetic events offers actionable therapeutic targets, improving patient outcomes through innovative treatments.
Area of Science:
- Oncology
- Genetics
- Pediatric Medicine
Background:
- Chromosomal fusions are increasingly recognized as critical molecular drivers in various solid tumors.
- Identification of these pathogenetic events in tumor specimens has become clinically actionable, particularly in rare pediatric cancers.
Purpose of the Study:
- To review oncogenic chromosomal rearrangements in pediatric solid tumors outside the central nervous system.
- To highlight the therapeutic potential and ongoing research for these rare tumor types.
Main Methods:
- Literature review of chromosomal rearrangements in pediatric solid tumors.
- Analysis of current and emerging therapeutic strategies targeting identified molecular drivers.
Main Results:
- Several pediatric solid tumors, including Ewing Sarcoma, Rhabdomyosarcoma, Fibrolamellar Hepatocellular Carcinoma, and Renal Cell Carcinoma, are characterized by driver gene fusions.
- A range of targeted therapeutics are being explored, including inhibitors for CDK4/6, FGFR, ALK, and others, alongside novel strategies like ONC201/TIC10.
Conclusions:
- Chromosomal fusions represent significant oncogenic events in pediatric solid tumors.
- Targeted therapies offer promising avenues for improving outcomes in these heterogeneous rare cancers, with ongoing innovation crucial for progress.
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