Glioblastoma multiforme (GBM) (GBM)

Glioblastoma multiforme (GBM) is the most aggressive and common primary malignant brain tumor in adults, characterized by high cellular heterogeneity and rapid proliferation [NCI]. These cells exhibit a high propensity for diffuse tissue invasion and extensive angiogenesis, which complicates surgical resection and localized treatment [StatPearls]. Molecularly, GBM is often driven by genetic alterations in signaling pathways such as EGFR, PI3K/AKT, and p53, alongside epigenetic modifications like MGMT promoter methylation [PubMed: 29406448]. Current pharmacological management primarily utilizes the DNA-alkylating agent temozolomide and the anti-VEGF monoclonal antibody bevacizumab [NEJM: 352:987-996]. However, therapeutic efficacy is severely hindered by the blood-brain barrier and the presence of treatment-resistant glioma stem cells [Nature Reviews Cancer: 13, 727–738]. Consequently, GBM remains a significant clinical challenge with a poor overall prognosis despite multimodal therapy.

Other names

GBMGlioblastomaGrade IV astrocytomaMalignant gliomaGlioblastoma, IDH-wildtype

Drugs targeting these cells primarily act through DNA alkylation to induce cell cycle arrest (e.g., temozolomide), inhibition of vascular endothelial growth factor (VEGF) to suppress angiogenesis (e.g., bevacizumab), or interference with intracellular signaling cascades such as the PI3K/AKT/mTOR pathway [StatPearls, PubMed: 15758009].