Introduction: Increasing attention has focused on the role of high-density lipoprotein function as a target for cardiprotection. Apolipoprotein A-I(Milano) (AIM) involves a single amino-acid mutation of the major wild-type protein carried on high-density lipoprotein (HDL) particles. Early evidence of beneficial activities of AIM has stimulated support in its development as a potential therapy to reduce cardiovascular risk.
Areas covered: The importance of HDL as a target and early data supporting the beneficial effects of AIM are reviewed. All clinical studies of AIM found in PubMed are reviewed.
Expert opinion: ETC-216 represents a lipid-deplete form of HDL containing recombinant AIM. While early evidence suggests that administration of ETC-216 promotes rapid regression of coronary atherosclerosis, bringing this compound to clinical practice will require further trials that evaluate its impact on cardiovascular events.