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New Approaches with Precision Medicine in Adult Brain Tumors
Annette Leibetseder1,2, Matthias Preusser3,4, Anna Sophie Berghoff3,4
1Department of Neurology 1, Kepler University Hospital, Johannes Kepler University Linz, 4020 Linz, Austria.
Abstract:
Primary central nervous system (CNS) tumors represent a heterogenous group of tumors. The 2021 fifth edition of the WHO Classification of Tumors of the CNS emphasizes the advanced role of molecular diagnostics with routine implementation of molecular biomarkers in addition to histologic features in the classification of CNS tumors. Thus, novel diagnostic methods such as DNA methylome profiling are increasingly used to provide a more precise diagnostic work-up of CNS tumors. In addition to these diagnostic precision medicine advantages, molecular alterations are also addressed therapeutically with targeted therapies. Like in other tumor entities, precision medicine has therefore also arrived in the treatment of CNS malignancies as the application of targeted therapies has shown promising response rates. Nevertheless, large prospective studies are currently missing as most targeted therapies were evaluated in single arm, basket, or platform trials. In this review, we focus on the current evidence of precision medicine in the treatment of primary CNS tumors in adults. We outline the pathogenic background and prevalence of the most frequent targetable genetic alterations and summarize the existing evidence of precision medicine approaches for the treatment of primary CNS tumors.
Insights
Precision medicine, utilizing targeted therapies based on molecular alterations, is transforming adult primary central nervous system (CNS) tumor treatment. This review examines current evidence for these advanced diagnostic and therapeutic strategies in CNS oncology.
Area of Science:
- Neuro-oncology
- Molecular Diagnostics
- Precision Medicine
Background:
- Primary central nervous system (CNS) tumors are diverse, with recent WHO classifications integrating molecular diagnostics alongside histology.
- Molecular biomarkers and advanced techniques like DNA methylome profiling enhance diagnostic accuracy for CNS tumors.
- Molecular alterations in CNS tumors are increasingly targeted by therapies, marking the arrival of precision medicine in neuro-oncology.
Purpose of the Study:
- To review the current evidence for precision medicine in treating adult primary CNS tumors.
- To outline the common targetable genetic alterations in CNS tumors and their prevalence.
- To summarize existing data on precision medicine approaches for primary CNS malignancies.
Main Methods:
- Literature review focusing on precision medicine in adult primary CNS tumors.
- Analysis of studies evaluating targeted therapies based on molecular alterations.
- Examination of diagnostic advancements including DNA methylome profiling.
Main Results:
- Targeted therapies show promising response rates in CNS malignancies.
- Most targeted therapies have been evaluated in early-phase or specialized trials, with a lack of large prospective studies.
- Identification and understanding of frequent targetable genetic alterations are crucial for treatment selection.
Conclusions:
- Precision medicine, guided by molecular diagnostics, offers a paradigm shift in treating primary CNS tumors.
- While promising, further large-scale prospective studies are needed to validate the efficacy of targeted therapies.
- Integrating molecular insights is essential for advancing the diagnosis and treatment of CNS tumors.
