Current status of precision oncology in adult glioblastoma

The concept of precision oncology, the application of targeted drugs based on comprehensive molecular profiling, has revolutionized treatment strategies in oncology. This review summarizes the current status of precision oncology in glioblastoma (GBM), the most common and aggressive primary brain tu...

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Main Authors: Johannes Weller, Anna‐Laura Potthoff, Thomas Zeyen, Christina Schaub, Cathrina Duffy, Matthias Schneider, Ulrich Herrlinger
Format: Article
Language:English
Published: Wiley 2024-12-01
Series:Molecular Oncology
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Online Access:https://doi.org/10.1002/1878-0261.13678
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author Johannes Weller
Anna‐Laura Potthoff
Thomas Zeyen
Christina Schaub
Cathrina Duffy
Matthias Schneider
Ulrich Herrlinger
author_facet Johannes Weller
Anna‐Laura Potthoff
Thomas Zeyen
Christina Schaub
Cathrina Duffy
Matthias Schneider
Ulrich Herrlinger
author_sort Johannes Weller
collection DOAJ
description The concept of precision oncology, the application of targeted drugs based on comprehensive molecular profiling, has revolutionized treatment strategies in oncology. This review summarizes the current status of precision oncology in glioblastoma (GBM), the most common and aggressive primary brain tumor in adults with a median survival below 2 years. Targeted treatments without prior target verification have consistently failed. Patients with BRAF V600E‐mutated GBM benefit from BRAF/MEK‐inhibition, whereas targeting EGFR alterations was unsuccessful due to poor tumor penetration, tumor cell heterogeneity, and pathway redundancies. Systematic screening for actionable molecular alterations resulted in low rates (< 10%) of targeted treatments. Efficacy was observed in one‐third and currently appears to be limited to BRAF‐, VEGFR‐, and mTOR‐directed treatments. Advancing precision oncology for GBM requires consideration of pathways instead of single alterations, new trial concepts enabling rapid and adaptive drug evaluation, a focus on drugs with sufficient bioavailability in the CNS, and the extension of target discovery and validation to the tumor microenvironment, tumor cell networks, and their interaction with immune cells and neurons.
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spelling doaj-art-e9a4c9ccc168445b969bf1b09603be972024-12-05T17:48:54ZengWileyMolecular Oncology1574-78911878-02612024-12-0118122927295010.1002/1878-0261.13678Current status of precision oncology in adult glioblastomaJohannes Weller0Anna‐Laura Potthoff1Thomas Zeyen2Christina Schaub3Cathrina Duffy4Matthias Schneider5Ulrich Herrlinger6Department of Neurooncology, Center for Neurology University Hospital Bonn GermanyDepartment of Neurosurgery University Hospital Bonn GermanyDepartment of Neurooncology, Center for Neurology University Hospital Bonn GermanyDepartment of Neurooncology, Center for Neurology University Hospital Bonn GermanyDepartment of Neurooncology, Center for Neurology University Hospital Bonn GermanyDepartment of Neurosurgery University Hospital Bonn GermanyDepartment of Neurooncology, Center for Neurology University Hospital Bonn GermanyThe concept of precision oncology, the application of targeted drugs based on comprehensive molecular profiling, has revolutionized treatment strategies in oncology. This review summarizes the current status of precision oncology in glioblastoma (GBM), the most common and aggressive primary brain tumor in adults with a median survival below 2 years. Targeted treatments without prior target verification have consistently failed. Patients with BRAF V600E‐mutated GBM benefit from BRAF/MEK‐inhibition, whereas targeting EGFR alterations was unsuccessful due to poor tumor penetration, tumor cell heterogeneity, and pathway redundancies. Systematic screening for actionable molecular alterations resulted in low rates (< 10%) of targeted treatments. Efficacy was observed in one‐third and currently appears to be limited to BRAF‐, VEGFR‐, and mTOR‐directed treatments. Advancing precision oncology for GBM requires consideration of pathways instead of single alterations, new trial concepts enabling rapid and adaptive drug evaluation, a focus on drugs with sufficient bioavailability in the CNS, and the extension of target discovery and validation to the tumor microenvironment, tumor cell networks, and their interaction with immune cells and neurons.https://doi.org/10.1002/1878-0261.13678biomarkerglioblastomamolecular profilingpersonalized medicineprecision oncologytargeted treatment
spellingShingle Johannes Weller
Anna‐Laura Potthoff
Thomas Zeyen
Christina Schaub
Cathrina Duffy
Matthias Schneider
Ulrich Herrlinger
Current status of precision oncology in adult glioblastoma
Molecular Oncology
biomarker
glioblastoma
molecular profiling
personalized medicine
precision oncology
targeted treatment
title Current status of precision oncology in adult glioblastoma
title_full Current status of precision oncology in adult glioblastoma
title_fullStr Current status of precision oncology in adult glioblastoma
title_full_unstemmed Current status of precision oncology in adult glioblastoma
title_short Current status of precision oncology in adult glioblastoma
title_sort current status of precision oncology in adult glioblastoma
topic biomarker
glioblastoma
molecular profiling
personalized medicine
precision oncology
targeted treatment
url https://doi.org/10.1002/1878-0261.13678
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