The GJA1 inhibitor clofazimine to treat glioblastoma

INDICATION: Brain cancer

Cell culture and mouse studies identified clofazimine as a GJA1 inhibitor that could help treat glioblastoma. Screening of a library of 727 FDA-approved small molecules in cell-based activity assays yielded clofazimine as the drug that most potently inhibited GJA1. In glioblastoma cancer stem cells from four patient-derived xenograft (PDX) specimens, the compound inhibited growth with IC₅₀ values of 0.99-2.02 μM. In a mouse model of glioblastoma, clofazimine decreased tumor growth compared with vehicle. Next steps could include optimizing clofazimine to improve solubility and blood-brain barrier (BBB) penetration...