Glioblastoma (GBM) stem cells have been implicated in tumour progression, treatment resistance and tumour recapitulation. Polson et al. therefore investigated the therapeutic effects of KHS101, which has been previously shown to promote neural differentiation. Unexpectedly, KHS101 was found to be cytotoxic in multiple patient-derived GBM cell lines, disrupting GBM cell energy metabolism and mitochondrial dynamics, promoting autophagy and apoptosis. In patient-derived xenograft mouse models of GBM, systemic KHS101 treatment reduced tumour growth and prolonged survival, without adverse effects.
References
Polson, E. et al. KHS101 disrupts energy metabolism in human glioblastoma cells and reduces tumor growth in mice. Sci. Transl Med. 10, eaar2718 (2018)10.1126/scitranslmed.aar2718
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Crunkhorn, S. Disrupting energy metabolism. Nat Rev Drug Discov 17, 708 (2018). https://doi.org/10.1038/nrd.2018.172
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DOI: https://doi.org/10.1038/nrd.2018.172
