However, it has only very limited efficacy.5 Currently, there is no targeted therapy available for this tumour type,6 largely due to the fact that molecular mechanisms underlying ICC development and progression remain poorly defined.8 Importantly, recent genomic studies have identified novel pathways and mutations in ICC, such as FGFR signalling and IDH1/2 mutations, which could be eventually targeted with specific drugs.9 However, the contribution of these genetic and signalling events in cholangiocarcinogenesis requires further validation using in vitro and in vivo approaches. This evidence concerns the gene IDH1 and intrahepatic cholangiocarcinoma.