Niemann-Pick C1-Like 1 (NPC1L1) is a protein found on the gastrointestinal tract's epithelial cells[5] as well as in hepatocytes.[6] Specifically, it appears to bind to a critical mediator of cholesterol absorption.
AliasesNPC1L1, NPC11L1, NPC1 like intracellular cholesterol transporter 1, SLC65A2, LDLCQ7 Quick facts Available structures, PDB ...
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The drug ezetimibe inhibits NPC1L1 causing a reduction in cholesterol absorption, resulting in a blood cholesterol reduction of 15-20%.[7] Polymorphic variations in the NPC1L1 gene could be associated with non-response to ezetimibe treatment.[8] One study found that people with inactivating mutations in the NPC1L1 gene had a lower LDL cholesterol level, as well as an around 50% reduction in the risk of coronary heart disease.[9]
NPC1L1 has been shown to be an accessory receptor for hepatitis C virus entry into cells, and thus ezetimibe might be used as a therapeutic strategy.[10]
As cancer appeared more frequently in patients treated with simvastatin-ezetimibe combination therapy in one clinical trial,[11] it had been hypothesized that NPC1L1 inhibition by ezetimibe might be associated with an increased cancer risk.[12] However, a meta-analysis of ezetimibe clinical data showed no increase in the risk of cancer from treatment with ezetimibe.[13]
