Applied Precision Cancer Medicine in Neuro-Oncology.

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.