Nifuroxazide (NFX) is a broad-spectrum antibacterial drug that has been used for the treatment of infectious diarrhoea since 1966. In 2008, the discovery of potent inhibition of the transcription factor signal transducer and activator of transcription STAT3 by NFX prompted studies as a potential anticancer agent. Subsequently, it was shown that NFX induces cancer cell apoptosis and inhibits tumour growth. Recently, NFX was identified as a potent inhibitor of aldehyde dehydrogenase ALDH1 that selectively kills ALDHhigh cancer-initiating cells. These two landmark discoveries - STAT3 and ALDH1 inhibition - strongly support the potential repositioning of NFX as a targeted anticancer agent. The related antiparasitic drug nifurtimox is undergoing clinical development for the treatment of paediatric tumours. The anticancer potential of NFX is highlighted here.
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