RESEARCH Novel anti-glioblastoma a r

Full list of author information is available at the end of the articleIntroduction Novel therapeutic options are sorely needed to target glioblastoma (GBM), a notoriously treatment-resistant brain cancer. GBM is a leading cause of cancer-related death in the pediatric and adult populations, with most patients succumbing within 1-2 years [1,2]. The standard therapies are inadequate, and their toxicities lead to severe life-long morbidity in the small number of patients that survive [2]. Despite this grim prognosis, GBM is an orphan disease that is in general not a priority for new drug development [3-5]. Moreover, the biology of GBM is complex and much remains to be learned about the putative key signaling pathways before they can be therapeutically exploited [6]. In view of the unmet and urgent clinical need, we were motivated to pursue recent data indicating that GBM may respond to some FDA-approved agents not previously identified as GBM therapeutics [7,8]. The in vitro screening of a broad range of drugs already approved for other indications is attractive as in vivo toxicity and pharmacology are well defined, and such compounds can enter GBM clinica