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Incorporating Targeted Therapy Into Neuro-Oncology Practice
Marjolein Geurts1, Juan B Blaquier2, Maarten Wijnenga3
1Departments of Neurology and Medical Oncology, Brain Tumor Center at Erasmus MC Cancer Institute, Rotterdam, the Netherlands.
Abstract:
The integration of targeted therapies into neuro-oncology is revolutionizing the management of primary CNS malignancies. Advances in sequencing technologies and the incorporation of molecular alterations into CNS tumor classification have led to more precise tumor prognosis and enabled the identification of actionable oncogenic drivers. However, challenges such as drug delivery, tumor and microenvironment heterogeneity, and limitations of preclinical models complicate the selection of effective therapies. This review presents a comprehensive framework for optimizing drug selection in neuro-oncology. We discuss strategies to enhance drug development and improve clinical trial success, including window-of-opportunity trials and advanced imaging techniques. Additionally, we highlight recent advances in the treatment of isocitrate dehydrogenase-mutant gliomas, focusing on the INDIGO study and its role in the regulatory approval of vorasidenib. The review also examines the use of MAPK inhibitors, from BRAF inhibitors to PAN-RAF inhibitors, in both pediatric and adult patients, as well as novel investigational agents. Finally, we explore emerging targeted therapies for rarer oncogenic drivers, such as FGFR and NTRK alterations, emphasizing the need for CNS-specific drug development strategies.
Insights
Targeted therapies are transforming neuro-oncology by identifying specific cancer drivers. This review outlines strategies to optimize drug selection and development for brain tumors, improving patient outcomes.
Area of Science:
- Neuro-oncology
- Molecular Oncology
- Translational Medicine
Background:
- Targeted therapies are revolutionizing primary central nervous system (CNS) malignancy management.
- Advances in molecular profiling enable precise prognosis and identification of oncogenic drivers.
- Challenges include drug delivery, tumor heterogeneity, and preclinical model limitations.
Purpose of the Study:
- To present a framework for optimizing targeted drug selection in neuro-oncology.
- To discuss strategies for enhancing drug development and clinical trial success.
- To review recent advances in targeted therapies for CNS malignancies.
Main Methods:
- Comprehensive literature review of targeted therapies in neuro-oncology.
- Analysis of strategies for drug development and clinical trial design.
- Examination of specific drug classes and oncogenic alterations.
Main Results:
- Highlighting the success of vorasidenib for isocitrate dehydrogenase-mutant gliomas (INDIGO study).
- Reviewing the application of MAPK inhibitors (BRAF, PAN-RAF) in pediatric and adult CNS tumors.
- Exploring emerging therapies for FGFR and NTRK alterations.
Conclusions:
- Optimizing drug selection requires addressing challenges in delivery and heterogeneity.
- CNS-specific drug development strategies are crucial for rare oncogenic drivers.
- Advancements in targeted therapies offer new hope for patients with primary CNS malignancies.
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