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Applied Precision Cancer Medicine in Neuro-Oncology
H Taghizadeh1,2, L Müllauer3, J Furtner2,4
1Department of Medicine I, Clinical Division of Oncology, Medical University of Vienna, Vienna, Austria.
Abstract:
Brain tumours that are refractory to treatment have a poor prognosis and constitute a major challenge in offering effective treatment strategies. By targeting molecular alterations, precision cancer medicine may be a viable option for the treatment of brain tumours. In this retrospective analysis of our PCM platform, we describe the molecular profiling of primary brain tumours from 50 patients. Tumour samples of the patients were examined by a 161-gene next-generation sequencing panel, immunohistochemistry, and fluorescence in situ hybridization (FISH). We identified 103 molecular aberrations in 36 (72%) of the 50 patients. The predominant mutations were TP53 (14.6%), IDH1 (9.7%) and PIK3CA (6.8%). No mutations were detected in 14 (28%) of the 50 patients. IHC demonstrated frequent overexpression of EGFR and mTOR, in 38 (76%) and 35 (70%) patients, respectively. Overexpression of PDGFRa and PDGFRb were less common and detected in 16 and four patients, respectively. For 35 patients a targeted therapy was recommended. In our database, the majority of patients displayed mutations, against which targeted therapy could be offered. Based on our observations, PCM may be a feasible novel treatment approach in neuro-oncology.
Insights
Precision cancer medicine (PCM) identified molecular targets in 72% of brain tumors. This approach offers a feasible treatment strategy for neuro-oncology patients with refractory tumors.
Area of Science:
- Neuro-oncology
- Genomics
- Precision Medicine
Background:
- Brain tumors refractory to treatment present significant challenges.
- Precision cancer medicine (PCM) offers a targeted approach to treatment.
- Molecular profiling is key to identifying actionable targets.
Purpose of the Study:
- To analyze the molecular profile of primary brain tumors.
- To assess the feasibility of PCM in neuro-oncology.
- To identify targetable molecular aberrations in brain tumor patients.
Main Methods:
- Retrospective analysis of the PCM platform.
- Molecular profiling using next-generation sequencing (161-gene panel), immunohistochemistry (IHC), and fluorescence in situ hybridization (FISH).
- Analysis of 50 primary brain tumor samples.
Main Results:
- 103 molecular aberrations identified in 72% of patients.
- Predominant mutations: TP53 (14.6%), IDH1 (9.7%), PIK3CA (6.8%).
- Frequent overexpression of EGFR (76%) and mTOR (70%) detected by IHC. Targeted therapy recommended for 35 patients.
Conclusions:
- The majority of brain tumor patients harbor mutations actionable by targeted therapies.
- PCM is a feasible and promising treatment approach in neuro-oncology.
- Molecular profiling is crucial for guiding treatment decisions in refractory brain tumors.
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