p97 Inhibitor CB-5083 Blocks ERAD in Trypanosoma brucei

Mol Biochem Parasitol. 2020 Sep:239:111313. doi: 10.1016/j.molbiopara.2020.111313. Epub 2020 Jul 28.

Abstract

Misfolded proteins trapped in the endoplasmic reticulum (ER) are specifically recognized and retrotranslocated to the cytosol by the ER-Associated Degradation (ERAD) system and delivered to the proteasome for destruction. This process was recently described in Trypanosoma brucei (T. brucei) using the misfolded epitope tagged Transferrin Receptor subunits ESAG7:Ty and HA:ESAG6 (HA:E6). Critical to this work was the proteasomal inhibitor MG132. However, MG132 has off-target inhibitory effects on lysosomal Cathepsin L that could cause misinterpretation of turnover results. Here, we evaluate an orally bioavailable p97 inhibitor, CB-5083, for use in T. brucei. p97 is a ubiquitous protein involved in many cellular events including the membrane extraction step of ERAD. CB-5083 strongly inhibits turnover of HA:E6, with comparable protein recovery to MG132 treatment. Interestingly, little deglycosylated cytoplasmic species accumulates, though it normally emerges with MG132 treatment. This suggests that CB-5083 blocks ERAD upstream of the proteasome, as expected for inhibition of the trypanosomal p97 orthologue TbVCP. Under CB-5083 treatment, HA:E6 is also strongly membrane-associated, suggesting ER localization. Finally, we provide an experimental example where CB-5083 treatment offers clarity to the off-target effects of MG132 treatment.

Keywords: CB-5083; ERAD; MG132; p97; trypanosome; turnover.

Publication types

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

MeSH terms

  • Adenosine Triphosphatases / antagonists & inhibitors
  • Cysteine Proteinase Inhibitors / pharmacology
  • Cytosol / metabolism
  • Endoplasmic Reticulum / metabolism
  • Endoplasmic Reticulum-Associated Degradation* / drug effects
  • Endoplasmic Reticulum-Associated Degradation* / physiology
  • Indoles / pharmacology*
  • Leupeptins / pharmacology*
  • Nuclear Proteins / antagonists & inhibitors
  • Proteasome Endopeptidase Complex / metabolism
  • Proteasome Inhibitors / pharmacology
  • Protein Folding
  • Protein Transport
  • Proteolysis / drug effects
  • Protozoan Proteins / metabolism
  • Pyrimidines / pharmacology*
  • Trypanosoma brucei brucei* / drug effects
  • Trypanosoma brucei brucei* / metabolism

Substances

  • CB-5083
  • Cysteine Proteinase Inhibitors
  • Indoles
  • Leupeptins
  • Nuclear Proteins
  • Proteasome Inhibitors
  • Protozoan Proteins
  • Pyrimidines
  • Proteasome Endopeptidase Complex
  • Adenosine Triphosphatases
  • p97 ATPase
  • benzyloxycarbonylleucyl-leucyl-leucine aldehyde