Molecularly Targeted Clinical Trials

Authors

Matthew A. Smith-Cohn, Neuro-Oncology Branch, National Cancer Institute, National Institutes of Health, 37 Convent Drive, Building 37, Room 1016, Bethesda, MD 20892, USA; Department of Neurology, The Johns Hopkins University School of Medicine, Baltimore, MD 21287, USA.Follow
Orieta Celiku, Neuro-Oncology Branch, National Cancer Institute, National Institutes of Health, 37 Convent Drive, Building 37, Room 1142, Bethesda, MD 20892, USA.
Mark R. Gilbert, Neuro-Oncology Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: Mark.Gilbert@nih.gov.

Document Type

Article

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.

Medical Subject Headings

Adult; Blood-Brain Barrier; Brain Neoplasms (drug therapy, genetics); Clinical Trials as Topic; Glioblastoma (drug therapy, genetics); Humans; Molecular Targeted Therapy

Publication Date

4-1-2021

Publication Title

Neurosurgery clinics of North America

E-ISSN

1558-1349

Volume

32

Issue

2

First Page

191

Last Page

210

PubMed ID

33781502

Digital Object Identifier (DOI)

10.1016/j.nec.2020.12.002

Recommended Citation

Smith-Cohn, Matthew A.; Celiku, Orieta; and Gilbert, Mark R., "Molecularly Targeted Clinical Trials" (2021). Neurology. 1749.
https://scholar.barrowneuro.org/neurology/1749