Feasibility of Targeting Glioblastoma Stem Cells: From Concept to Clinical Trials

Curr Top Med Chem. 2019;19(32):2974-2984. doi: 10.2174/1568026619666191112140939.


Objective: Glioblastoma is a highly aggressive and invasive brain and Central Nervous System (CNS) tumor. Current treatment options do not prolong overall survival significantly because the disease is highly prone to relapse. Therefore, research to find new therapies is of paramount importance. It has been discovered that glioblastomas contain a population of cells with stem-like properties and that these cells are may be responsible for tumor recurrence.

Methods: A review of relevant papers and clinical trials in the field was conducted. A PubMed search with related keywords was used to gather the data. For example, "glioblastoma stem cells AND WNT signaling" is an example used to find information on clinical trials using the database ClinicalTrials.gov.

Results: Cancer stem cell research has several fundamental issues and uncertainties that should be taken into consideration. Theoretically, a number of treatment options that target glioblastoma stem cells are available for patients. However, only a few of them have obtained promising results in clinical trials. Several strategies are still under investigation.

Conclusion: The majority of treatments to target cancer stem cells have failed during clinical trials. Taking into account a number of biases in the field and the number of unsuccessful investigations, the application of the cancer stem cells concept is questionable in clinical settings, at least with respect to glioblastoma.

Keywords: Brain tumor; CAR T-cell therapy; Cancer stem cells; Glioblastoma; Molecular targeted therapy; Stem cell self-renewal; Vaccine therapy..

Publication types

  • Review

MeSH terms

  • Brain Neoplasms / metabolism
  • Brain Neoplasms / pathology*
  • Clinical Trials as Topic*
  • Feasibility Studies
  • Glioblastoma / metabolism
  • Glioblastoma / pathology*
  • Humans
  • Immunotherapy
  • Neoplastic Stem Cells / drug effects*
  • Neoplastic Stem Cells / metabolism
  • Signal Transduction