Molecularly Targeted Clinical Trials

Neurosurg Clin N Am. 2021 Apr;32(2):191-210. doi: 10.1016/ Epub 2021 Feb 18.


Glioblastoma remains incurable despite advances in surgery, radiation, and chemotherapy, underscoring the need for new therapies. The genetic heterogenicity, presence of redundant molecular pathways, and the blood-brain barrier have limited the applicability of molecularly targeted agents. The therapeutic benefit seen with a small subset of patients suggests, however, that patient selection is critical. Recent investigations show that molecularly targeted synthetic lethality is a promising complementary approach. The article provides an overview of the challenges of molecularly targeted therapy in adults with glioblastoma, including current trials and future therapeutic directions.

Keywords: Blood-brain barrier; Glioblastoma; Heterogenicity; Synthetic lethality; Targeted therapy.

Publication types

  • Review

MeSH terms

  • Adult
  • Blood-Brain Barrier
  • Brain Neoplasms* / drug therapy
  • Brain Neoplasms* / genetics
  • Clinical Trials as Topic
  • Glioblastoma* / drug therapy
  • Glioblastoma* / genetics
  • Humans
  • Molecular Targeted Therapy