Comprehensive genetic profiling and molecularly guided treatment for patients with primary CNS tumors

Despite major advances in molecular profiling and classification of primary brain tumors, personalized treatment remains limited for most patients. Here, we explored the feasibility of individual molecular profiling and the efficacy of biomarker-guided therapy for adult patients with primary brain c...

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Bibliographic Details
Published in:NPJ precision oncology 2024-08, Vol.8 (1), p.180-11
Main Authors: Kuehn, Julia C., Metzger, Patrick, Neidert, Nicolas, Matysiak, Uta, Gräßel, Linda, Philipp, Ulrike, Bleul, Sabine, Pauli, Thomas, Falkenstein, Julia, Bertemes, Henriette, Cysar, Stepan, Hess, Maria Elena, Frey, Anna Verena, Duque-Afonso, Jesús, Schorb, Elisabeth, Machein, Marcia, Beck, Jürgen, Schnell, Oliver, von Bubnoff, Nikolas, Illert, Anna L., Peters, Christoph, Brummer, Tilman, Prinz, Marco, Miething, Cornelius, Becker, Heiko, Lassmann, Silke, Werner, Martin, Börries, Melanie, Duyster, Justus, Heiland, Dieter H., Sankowski, Roman, Scherer, Florian
Format: Article
Language:English
Subjects:
692/4017
692/4028/67/1922
692/53/2421
692/699/67/1922
Biomarkers
Brain cancer
Brain research
Cancer Research
Cell cycle
Clinical outcomes
Clinical trials
Gene Therapy
Glioma
Human Genetics
Internal Medicine
Medical research
Medicine
Medicine & Public Health
Neurosurgery
Observational studies
Oncology
Precision medicine
Tumors
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Summary:Despite major advances in molecular profiling and classification of primary brain tumors, personalized treatment remains limited for most patients. Here, we explored the feasibility of individual molecular profiling and the efficacy of biomarker-guided therapy for adult patients with primary brain cancers in the real-world setting within the molecular tumor board Freiburg, Germany. We analyzed genetic profiles, personalized treatment recommendations, and clinical outcomes of 102 patients with 21 brain tumor types. Alterations in the cell cycle, BRAF, and mTOR pathways most frequently led to personalized treatment recommendations. Molecularly informed therapies were recommended in 71% and implemented in 32% of patients with completed molecular diagnostics. The disease control rate following targeted treatment was 50% and the overall response rate was 30%, with a progression-free survival 2/1 ratio of at least 1.3 in 31% of patients. This study highlights the efficacy of molecularly guided treatment and the need for biomarker-stratified trials in brain cancers.
ISSN:2397-768X
2397-768X
DOI:10.1038/s41698-024-00674-y