Fighting neurodegeneration with rapamycin: mechanistic insights

Nat Rev Neurosci. 2011 Jul 20;12(8):437-52. doi: 10.1038/nrn3068.


A growing number of studies point to rapamycin as a pharmacological compound that is able to provide neuroprotection in several experimental models of neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, Huntington's disease and spinocerebellar ataxia type 3. In addition, rapamycin exerts strong anti-ageing effects in several species, including mammals. By inhibiting the activity of mammalian target of rapamycin (mTOR), rapamycin influences a variety of essential cellular processes, such as cell growth and proliferation, protein synthesis and autophagy. Here, we review the molecular mechanisms underlying the neuroprotective effects of rapamycin and discuss the therapeutic potential of this compound for neurodegenerative diseases.

Publication types

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

MeSH terms

  • Humans
  • Nerve Degeneration / drug therapy*
  • Nerve Degeneration / metabolism
  • Neurodegenerative Diseases / drug therapy*
  • Neurodegenerative Diseases / metabolism
  • Signal Transduction / drug effects
  • Signal Transduction / physiology
  • Sirolimus / pharmacokinetics
  • Sirolimus / therapeutic use*
  • TOR Serine-Threonine Kinases / metabolism*


  • TOR Serine-Threonine Kinases
  • Sirolimus