Glioblastoma multiforme (GBM) is the most commonly diagnosed primary Central Nervous System tumors in
adults, with approximately 10,000 new cases annually in the United States. GBMs rank among the deadliest of all human
cancers with no curative options available; no meaningful therapeutic advances have been made in over 30 years.
Recurrence is inevitable in the vast majority of cases, and a permanent cure for GBM remains elusive. Even those
individuals who respond to first-line therapies subsequently exhibit a limited response to second-line therapies, which
eventually leads to their demise. Therefore, there is an urgent need to develop novel therapeutic options that effectively
target therapy-resistant GBM cells. GBM stem cells (GSCs) are a subpopulation of highly tumorigenic cells that contain
stem cell characteristics. While our understanding of GSCs is evolving, studies support that GSCs drive GBM propagation
and enable resistance to conventional therapies such as radiation. Identification of novel therapeutics designed to target
the GSC population in GBM has emerged as a promising strategy to overcome this lethal disease. This review summarizes
the latest understanding about translational research toward molecular characterization of GSCs and potential therapeutic
application of the recent pre-clinical research discoveries to the clinic.
Keywords: Glioma, glioblastoma, cancer stem cell, therapeutic development, experimental therapeutics, Malignant brain tumors, glioblastoma or GBM)
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