Abstract

Intracranial tumors encompass a heterogeneous group of neoplasms, including gliomas, meningiomas, pituitary adenomas, schwannomas, craniopharyngiomas, ependymomas, medulloblastomas, and primary central nervous system lymphomas. These tumors present significant challenges due to their diverse molecular characteristics, critical locations, and the unique obstacles posed by the blood-brain barrier (BBB) and blood-tumor barrier (BTB), which limit the efficacy of systemic therapies. Recent advances in molecular biology and genomics have enabled the identification of specific molecular pathways and targets, paving the way for innovative precision therapies. This review examines the current state of targeted therapies for intracranial tumors, including receptor tyrosine kinase (RTK) inhibitors, PI3K/AKT/mTOR inhibitors, RAF/MEK/ERK pathway inhibitors, IDH mutation inhibitors, immune checkpoint inhibitors, and CAR-T cell therapies. Emphasis is placed on the role of the BBB and BTB in modulating drug delivery and therapeutic outcomes. Strategies to overcome these barriers, such as focused ultrasound, nanoparticle-based delivery systems, and convection-enhanced delivery, are also explored. Furthermore, the manuscript reviews clinical trial data, highlighting successes and limitations across different tumor types. It delves into emerging therapeutic approaches, including combination of regimens and personalized treatments based on molecular profiling. By synthesizing the latest research, this article aims to provide a comprehensive understanding of the advancements and ongoing challenges in the targeted treatment of intracranial tumors. The findings underscore the necessity for innovative delivery systems and more extensive clinical trials to optimize therapeutic strategies. This review aspires to inform future research and clinical practices, aiming to improve patient outcomes and quality of life in the management of these complex and life-threatening conditions.