De novo lipogenesis is essential for platelet production in humans

Nat Metab. 2020 Oct;2(10):1163-1178. doi: 10.1038/s42255-020-00272-9. Epub 2020 Sep 14.

Abstract

Acetyl-CoA carboxylase (ACC) catalyses the first step of de novo lipogenesis (DNL). Pharmacologic inhibition of ACC has been of interest for therapeutic intervention in a wide range of diseases. We demonstrate here that ACC and DNL are essential for platelet production in humans and monkeys, but in not rodents or dogs. During clinical evaluation of a systemically distributed ACC inhibitor, unexpected dose-dependent reductions in platelet count were observed. While platelet count reductions were not observed in rat and dog toxicology studies, subsequent studies in cynomolgus monkeys recapitulated these platelet count reductions with a similar concentration response to that in humans. These studies, along with ex vivo human megakaryocyte maturation studies, demonstrate that platelet lowering is a consequence of DNL inhibition likely to result in impaired megakaryocyte demarcation membrane formation. These observations demonstrate that while DNL is a minor quantitative contributor to global lipid balance in humans, DNL is essential to specific lipid pools of physiological importance.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetyl-CoA Carboxylase / antagonists & inhibitors
  • Acetyl-CoA Carboxylase / metabolism
  • Animals
  • Blood Platelets*
  • Diabetes Mellitus, Type 2 / drug therapy
  • Dogs
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Enzyme Inhibitors / pharmacology
  • Gene Expression / drug effects
  • Humans
  • Lipid Metabolism
  • Lipogenesis / physiology*
  • Macaca fascicularis
  • Megakaryocytes / physiology
  • Platelet Count
  • Rats

Substances

  • Enzyme Inhibitors
  • Acetyl-CoA Carboxylase