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Bile acids (BAs) are amphiphilic molecules traditionally recognized for their role in lipid digestion but have gained increased interest for their therapeutic potential. Among them, ursodeoxycholic acid (UDCA) is the most widely prescribed and has been FDA-approved in the treatment of primary biliary cholangitis (PBC), the most common chronic cholestatic liver disease, while also being used off-label in multiple other disorders. The therapeutic effects of BAs are linked to their capacity to modulate signaling pathways, reduce hepatocellular injury, and regulate inflammation. Their physicochemical properties, particularly hydrophobicity, influence both efficacy and toxicity, of which the mechanisms involving receptors such as farnesoid X receptor (FXR), vitamin D receptor (VDR) and Takeda G protein-coupled receptor 5 (TGR5) help to explain. Recent regulatory milestones include the FDA-approval of chenodeoxycholic acid (CDCA) in the treatment of cerebrotendinous xanthomatosis (CTX) and ongoing clinical trials such as that of norucholic acid (NCA) in the treatment of primary sclerosing cholangitis (PSC). Expanding research is redefining the BA therapeutic landscape, with applications spanning cholestatic, metabolic, and neurodegenerative diseases. This review will explore established and emerging BA-based monotherapies, combination regimens, and novel BA-driven drug delivery systems.
Vu et al. (Mon,) studied this question.