Anti-tumor efficacy and safety of AEV01 in preclinical glioblastoma and hepatocellular carcinoma models

Glioblastoma (GB) and hepatocellular carcinoma (HCC) are aggressive cancers with poor prognoses, often leading to less than a year of survival. Therapeutic resistance underscores the need for novel therapeutic strategies. AEV01, a specialized lipophilic extract derived from the roots of Picrorhiza kurroa, has shown promise as a potential anti-cancer agent. In this context, the current study aimed to evaluate the anti-tumor efficacy and safety profile of AEV01 both in vitro and in vivo in GB and HCC models. Briefly, cytotoxicity and apoptosis were assessed using MTT assay and AO/EtBr staining, while ELISA and immunofluorescence measured inflammatory markers like IL-6, IL-18, IL-1β, TNF-α, CASP-1, CRP, TP53 and CD36 expression. In vivo, ELISA was performed against the inflammatory and tumor suppressor markers while, histopathological analysis assessed tumor morphology and organ toxicity. AEV01 exhibited dose-dependent cytotoxicity against U-87 MG glioblastoma and HepG2 liver cancer cells, with optimal concentrations at 400 µg/ml and 300 µg/ml respectively. Treatment downregulated inflammatory markers, CD36 expression and concomitantly increased TP53 expression. Xenograft models depicted similar results, with reduced tumor markers expression, reduced changes in tissue architecture, and no significant organ toxicity. Thus, AEV01 demonstrated potent anti-tumor activity with a favorable safety profile, suggesting its potential as a novel therapeutic agent for Glioblastoma and HCC, warranting further clinical investigation.