A small molecule Smac mimic potentiates TRAIL- and TNFalpha-mediated cell death

Science. 2004 Sep 3;305(5689):1471-4. doi: 10.1126/science.1098231.

Abstract

We describe the synthesis and properties of a small molecule mimic of Smac, a pro-apoptotic protein that functions by relieving inhibitor-of-apoptosis protein (IAP)-mediated suppression of caspase activity. The compound binds to X chromosome- encoded IAP (XIAP), cellular IAP 1 (cIAP-1), and cellular IAP 2 (cIAP-2) and synergizes with both tumor necrosis factor alpha (TNFalpha) and TNF-related apoptosis-inducing ligand (TRAIL) to potently induce caspase activation and apoptosis in human cancer cells. The molecule has allowed a temporal, unbiased evaluation of the roles that IAP proteins play during signaling from TRAIL and TNF receptors. The compound is also a lead structure for the development of IAP antagonists potentially useful as therapy for cancer and inflammatory diseases.

Publication types

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

MeSH terms

  • Alkynes / chemical synthesis
  • Alkynes / chemistry
  • Alkynes / metabolism
  • Alkynes / pharmacology*
  • Apoptosis Regulatory Proteins
  • Apoptosis*
  • Biotinylation
  • Carrier Proteins* / chemistry
  • Carrier Proteins* / metabolism
  • Caspase Inhibitors
  • Caspases / metabolism
  • Cell Line, Tumor
  • Computer Simulation
  • Dimerization
  • Dipeptides / chemical synthesis
  • Dipeptides / chemistry
  • Dipeptides / metabolism
  • Dipeptides / pharmacology*
  • Diynes
  • Glioblastoma
  • Humans
  • Inhibitor of Apoptosis Proteins
  • Intracellular Signaling Peptides and Proteins
  • Membrane Glycoproteins / metabolism
  • Membrane Glycoproteins / pharmacology*
  • Mitochondrial Proteins* / chemistry
  • Mitochondrial Proteins* / metabolism
  • Molecular Mimicry*
  • NF-kappa B / metabolism
  • Poly(ADP-ribose) Polymerases / metabolism
  • Protein Binding
  • Protein Conformation
  • Protein Engineering
  • Proteins / metabolism
  • Signal Transduction
  • TNF-Related Apoptosis-Inducing Ligand
  • Tetrazoles / chemical synthesis
  • Tetrazoles / chemistry
  • Tetrazoles / metabolism
  • Tetrazoles / pharmacology*
  • Tumor Necrosis Factor-alpha / metabolism
  • Tumor Necrosis Factor-alpha / pharmacology*
  • X-Linked Inhibitor of Apoptosis Protein

Substances

  • 1,6-bis(4,7-diaza-6-methyl-5-oxo-3-((2-((5-(phenylthio)tetrazol-1-yl)methyl)pyrrolidin-1-yl)carbonyl)octyloxy)-2,4-hexadiyne
  • Alkynes
  • Apoptosis Regulatory Proteins
  • Carrier Proteins
  • Caspase Inhibitors
  • DIABLO protein, human
  • Dipeptides
  • Diynes
  • Inhibitor of Apoptosis Proteins
  • Intracellular Signaling Peptides and Proteins
  • Membrane Glycoproteins
  • Mitochondrial Proteins
  • NF-kappa B
  • Proteins
  • RMT5265
  • TNF-Related Apoptosis-Inducing Ligand
  • TNFSF10 protein, human
  • Tetrazoles
  • Tumor Necrosis Factor-alpha
  • X-Linked Inhibitor of Apoptosis Protein
  • XIAP protein, human
  • Poly(ADP-ribose) Polymerases
  • Caspases