Pharmacologic Agents Targeting Autophagy

J Clin Invest. 2015 Jan;125(1):5-13. doi: 10.1172/JCI73937. Epub 2015 Jan 2.


Autophagy is an important intracellular catabolic mechanism critically involved in regulating tissue homeostasis. The implication of autophagy in human diseases and the need to understand its regulatory mechanisms in mammalian cells have stimulated research efforts that led to the development of high-throughput screening protocols and small-molecule modulators that can activate or inhibit autophagy. Herein we review the current landscape in the development of screening technology as well as the molecules and pharmacologic agents targeting the regulatory mechanisms of autophagy. We also evaluate the potential therapeutic application of these compounds in different human pathologies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Autophagy / drug effects*
  • Calcium Signaling
  • Enzyme Inhibitors / pharmacology
  • Humans
  • Phosphoinositide-3 Kinase Inhibitors
  • Phosphoric Monoester Hydrolases / antagonists & inhibitors
  • Sirolimus / pharmacology
  • TOR Serine-Threonine Kinases / antagonists & inhibitors
  • Vacuolar Proton-Translocating ATPases / antagonists & inhibitors


  • Enzyme Inhibitors
  • Phosphoinositide-3 Kinase Inhibitors
  • MTOR protein, human
  • TOR Serine-Threonine Kinases
  • Phosphoric Monoester Hydrolases
  • myo-inositol-1 (or 4)-monophosphatase
  • Vacuolar Proton-Translocating ATPases
  • Sirolimus