Isoform-specific inhibitors of ACATs: recent advances and promising developments

Future Med Chem. 2011 Dec;3(16):2039-61. doi: 10.4155/fmc.11.158.

Abstract

Acyl-CoA:cholesterol acyltransferase (ACAT) is a promising therapeutic target for cardiovascular diseases. Although a number of synthetic ACAT inhibitors have been developed, they have failed to show efficacy in clinical trials. Now, the presence of two ACAT isoforms with distinct functions, ACAT1 and ACAT2, has been discovered. Thus, the selectivity of ACAT inhibitors toward the two isoforms is important for their development as novel anti-atherosclerotic agents. The selectivity study indicated that fungal pyripyropene A (PPPA) is only an ACAT2-specific inhibitor. Furthermore, PPPA proved orally active in atherogenic mouse models, indicating it possessed cholesterol-lowering and atheroprotective activities. Certain PPPA derivatives, semi-synthetically prepared, possessed more potent and selective in vitro activity than PPPA against ACAT2. This review covers these studies and describes the future prospects of ACAT2-specific inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Enzyme Inhibitors / pharmacology*
  • Inhibitory Concentration 50
  • Isoenzymes / antagonists & inhibitors*
  • Mice
  • Mice, Knockout
  • Sterol O-Acyltransferase / antagonists & inhibitors*

Substances

  • Enzyme Inhibitors
  • Isoenzymes
  • Sterol O-Acyltransferase