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Review
. 2021 Apr;32(2):191-210.
doi: 10.1016/j.nec.2020.12.002. Epub 2021 Feb 18.

Molecularly Targeted Clinical Trials

Matthew A Smith-Cohn  1 , Orieta Celiku  2 , Mark R Gilbert  3
Affiliations
Review

Molecularly Targeted Clinical Trials

Matthew A Smith-Cohn et al. Neurosurg Clin N Am. 2021 Apr.

Abstract

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.

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Conflict of interest statement

Disclosure The authors have nothing to disclose.