Molecularly driven therapies in the treatment of primary brain tumors.

Abstract

Molecular profiling has revolutionized the diagnosis, classification, and treatment of various cancers, with advances in next-generation sequencing and DNA methylation profiling offering unprecedented insights into tumor biology. This paradigm shift has enhanced the understanding of driver mutations in cancers translating into improved patient outcomes. In primary brain tumors, particularly gliomas, molecular profiling has redefined classification frameworks, yet meaningful improvements in patient survival remain elusive, particularly for glioblastoma. However, recent strides in molecularly targeted therapies have led to landmark FDA approvals, including agents such as vorasidenib for IDH-mutant gliomas, the combination of dabrafenib trametinib for BRAF mutated tumors, and TRK inhibitors for NTRK fusion-positive tumors. While conventional treatments like surgery, radiation, and chemotherapy remain the standard of care for gliomas, the integration of molecular-driven therapies is beginning to shape clinical management strategies. This article explores the evolving role of molecularly targeted treatments in adult primary brain tumors, examining their current applications and future potential.