Anticancer actions of lysosomally targeted inhibitor, LCL521, of acid ceramidase

PLoS One. 2017 Jun 14;12(6):e0177805. doi: 10.1371/journal.pone.0177805. eCollection 2017.

Abstract

Acid ceramidase, which catalyzes ceramide hydrolysis to sphingosine and free fatty acid mainly in the lysosome, is being recognized as a potential therapeutic target for cancer. B13 is an effective and selective acid ceramidase inhibitor in vitro, but not as effective in cells due to poor access to the lysosomal compartment. In order to achieve targeting of B13 to the lysosome, we designed lysosomotropic N, N-dimethyl glycine (DMG)-conjugated B13 prodrug LCL521 (1,3-di-DMG-B13). Our previous results indicated the efficient delivery of B13 to the lysosome resulted in augmented effects of LCL521 on cellular acid ceramidase as evaluated by effects on substrate/product levels. Our current studies indicate that functionally, this translated into enhanced inhibition of cell proliferation. Moreover, there were greater synergistic effects of LCL521 with either ionizing radiation or Tamoxifen. Taken together, these results clearly indicate that compartmental targeting for the inhibition of acid ceramidase is an efficient and valuable therapeutic strategy.

MeSH terms

  • Acid Ceramidase / antagonists & inhibitors*
  • Antineoplastic Agents / chemical synthesis*
  • Antineoplastic Agents / chemistry
  • Antineoplastic Agents / pharmacology
  • Breast Neoplasms / drug therapy
  • Breast Neoplasms / enzymology*
  • Breast Neoplasms / radiotherapy
  • Cell Proliferation / drug effects
  • Cell Proliferation / radiation effects
  • Cell Survival / drug effects
  • Cell Survival / radiation effects
  • Drug Synergism
  • Female
  • Humans
  • Nitrobenzenes / chemistry*
  • Prodrugs / chemical synthesis*
  • Prodrugs / chemistry
  • Prodrugs / pharmacology
  • Propanolamines / chemistry*
  • Tamoxifen / pharmacology

Substances

  • Antineoplastic Agents
  • DMG-B13
  • Nitrobenzenes
  • Prodrugs
  • Propanolamines
  • Tamoxifen
  • Acid Ceramidase