A novel rapamycin analog is highly selective for mTORC1 in vivo

Nat Commun. 2019 Jul 19;10(1):3194. doi: 10.1038/s41467-019-11174-0.

Abstract

Rapamycin, an inhibitor of mechanistic Target Of Rapamycin Complex 1 (mTORC1), extends lifespan and shows strong potential for the treatment of age-related diseases. However, rapamycin exerts metabolic and immunological side effects mediated by off-target inhibition of a second mTOR-containing complex, mTOR complex 2. Here, we report the identification of DL001, a FKBP12-dependent rapamycin analog 40x more selective for mTORC1 than rapamycin. DL001 inhibits mTORC1 in cell culture lines and in vivo in C57BL/6J mice, in which DL001 inhibits mTORC1 signaling without impairing glucose homeostasis and with substantially reduced or no side effects on lipid metabolism and the immune system. In cells, DL001 efficiently represses elevated mTORC1 activity and restores normal gene expression to cells lacking a functional tuberous sclerosis complex. Our results demonstrate that highly selective pharmacological inhibition of mTORC1 can be achieved in vivo, and that selective inhibition of mTORC1 significantly reduces the side effects associated with conventional rapalogs.

Publication types

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

MeSH terms

  • Animals
  • Cell Line
  • Drug Discovery
  • Gene Expression / drug effects
  • Humans
  • Immune System / drug effects
  • Lipid Metabolism / drug effects
  • Mechanistic Target of Rapamycin Complex 1 / antagonists & inhibitors*
  • Mice
  • Mice, Inbred C57BL
  • Proteomics
  • Signal Transduction / drug effects
  • Sirolimus / analogs & derivatives*
  • Sirolimus / chemistry
  • Sirolimus / pharmacology*
  • TOR Serine-Threonine Kinases
  • Tuberous Sclerosis

Substances

  • Mechanistic Target of Rapamycin Complex 1
  • TOR Serine-Threonine Kinases
  • Sirolimus