Emerging therapies for glioblastoma Journal Article


Authors: Thomas, A. A.; Brennan, C. W.; Deangelis, L. M.; Omuro, A. M.
Article Title: Emerging therapies for glioblastoma
Abstract: IMPORTANCE: Glioblastoma is the most common primary malignant brain tumor, but despite multimodal treatment with surgery, radiotherapy, and temozolomide chemotherapy, the prognosis is poor, with a median survival of 16 to 19 months and poor quality of life throughout the disease course. New treatments are needed. EVIDENCE REVIEW: Articles were identified through a search of PubMed references from March 2005 through January 2014, using the terms glioblastoma, glioma, malignant glioma, and brain neoplasm, as well as by search of the authors' files. Clinical trials were identified in the Clinicaltrials.gov registry. FINDINGS: Advances in the understanding of the molecular biology of glioblastoma are being rapidly translated into innovative clinical trials, capitalizing on improved genomic, epigenetic, transcriptional, and proteomic characterization of glioblastomas as well as host factors, including the brain microenvironment and immune system interactions. Therapies targeting tumor growth factor receptors and downstream pathways, angiogenesis, modulation of cancer stemlike cells, cell cycle regulation, oncolytic viruses, new radiotherapy techniques, and immunotherapy, including vaccines and modulation of immune checkpoints (eg, programmed cell death 1 and cytotoxic T-lymphocyte antigen 4), are under investigation. In addition to novel agents, techniques to circumvent the blood-brain barrier to facilitate central nervous system drug exposure are being developed. CONCLUSIONS AND RELEVANCE: Glioblastoma is an aggressive tumor with heterogeneous molecular features and complex host interactions, many of which are amenable to therapeutic intervention. Meaningful treatment advances will depend on identifying agents that target mechanistic vulnerabilities that are relevant to specific subgroups of patients; increasing patient enrollment into clinical trials is essential to accelerate the development of patient-tailored treatments.
Journal Title: JAMA Neurology
Volume: 71
Issue: 11
ISSN: 2168-6149
Publisher: American Medical Association  
Date Published: 2014-11-01
Start Page: 1437
End Page: 1444
Language: English
DOI: 10.1001/jamaneurol.2014.1701
PROVIDER: scopus
PUBMED: 25244650
DOI/URL:
Notes: Export Date: 2 January 2015 -- Source: Scopus
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MSK Authors
  1. Cameron Brennan
    226 Brennan
  2. Antonio Marcilio Padula Omuro
    204 Omuro
  3. Alissa A Thomas
    17 Thomas
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