Comprehensive genetic profiling and molecularly guided treatment for patients with primary CNS tumors
Julia C Kuehn1,2, Patrick Metzger3,4, Nicolas Neidert2,5,6
1Department of Medicine I, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg, Freiburg, Germany.
Abstract:
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.
Insights
Individual molecular profiling can guide brain cancer treatment. Biomarker-guided therapies showed promising efficacy, with 30% overall response rates and 50% disease control in real-world settings.
Area of Science:
- Oncology
- Genetics
- Neuro-oncology
Background:
- Personalized treatment for primary brain tumors is limited despite advances in molecular profiling.
- Real-world data on biomarker-guided therapy efficacy in adult brain cancers is needed.
Purpose of the Study:
- To assess the feasibility of individual molecular profiling for adult brain cancer patients.
- To evaluate the efficacy of biomarker-guided therapy in a real-world setting.
- To identify frequently altered pathways guiding treatment recommendations.
Main Methods:
- Analysis of genetic profiles, treatment recommendations, and clinical outcomes for 102 adult patients with 21 brain tumor types.
- Retrospective review of data from the molecular tumor board Freiburg.
- Correlation of molecular alterations with treatment response and survival.
Main Results:
- Personalized treatment recommendations were driven by alterations in cell cycle, BRAF, and mTOR pathways.
- Molecularly informed therapies were recommended in 71% and implemented in 32% of patients.
- Targeted treatment achieved a 50% disease control rate and 30% overall response rate, with 31% achieving a progression-free survival ratio of at least 1.3.
Conclusions:
- Molecularly guided treatment is effective for brain cancers in a real-world setting.
- Biomarker-stratified clinical trials are essential for advancing brain cancer therapy.
- Individual molecular profiling can inform personalized treatment strategies for brain tumor patients.
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