Carfilzomib resistance due to ABCB1/MDR1 overexpression is overcome by nelfinavir and lopinavir in multiple myeloma

Leukemia. 2018 Feb;32(2):391-401. doi: 10.1038/leu.2017.212. Epub 2017 Jul 5.

Abstract

Proteasome inhibitor (PI) carfilzomib (CFZ) has activity superior to bortezomib (BTZ) and is increasingly incorporated in multiple myeloma (MM) frontline therapy and relapsed settings. Most MM patients ultimately experience PI-refractory disease, an unmet medical need with poorly understood biology and dismal outcome. Pharmacologic targeting of ABCB1 improved patient outcomes, including MM, but suffered from adverse drug effects and insufficient plasma concentrations. Proteomics analysis identified ABCB1 overexpression as the most significant change in CFZ-resistant MM cells. We addressed the functional role of ABCB1 overexpression in MM and observed significantly upregulated ABCB1 in peripheral blood malignant plasma cells (PCs) vs untreated patients' bone marrow PC. ABCB1 overexpression reduces the proteasome-inhibiting activity of CFZ due to drug efflux, in contrast to BTZ. Likewise, the cytotoxicity of established anti-MM drugs was significantly reduced in ABCB1-expressing MM cells. In search for potential drugs targeting ABCB1 in clinical trials, we identified the HIV protease inhibitors nelfinavir (NFV) and lopinavir (LPV) as potent functional modulators of ABCB1-mediated drug export, most likely via modulation of mitochondria permeability transition pore. NFV and LPV restored CFZ activity at therapeutically relevant drug levels and thus represent ready-to-use drugs to be tested in clinical trials to target ABCB1 and to re-sensitize PC to established myeloma drugs, in particular CFZ.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily B / genetics
  • Antineoplastic Agents / pharmacology
  • Bortezomib / pharmacology
  • Cell Line, Tumor
  • Drug Resistance, Neoplasm / genetics*
  • HIV Protease Inhibitors / pharmacology
  • Humans
  • Lopinavir / pharmacology*
  • Multiple Myeloma / drug therapy*
  • Multiple Myeloma / genetics*
  • Nelfinavir / pharmacology*
  • Oligopeptides / pharmacology*
  • Plasma Cells / drug effects
  • Proteasome Endopeptidase Complex / drug effects
  • Proteasome Endopeptidase Complex / genetics
  • Proteasome Inhibitors / pharmacology

Substances

  • ABCB1 protein, human
  • ATP Binding Cassette Transporter, Subfamily B
  • Antineoplastic Agents
  • HIV Protease Inhibitors
  • Oligopeptides
  • Proteasome Inhibitors
  • Lopinavir
  • Bortezomib
  • carfilzomib
  • Proteasome Endopeptidase Complex
  • Nelfinavir