Combination of l-Carnitine with Lipophilic Linkage-Donating Gemcitabine Derivatives as Intestinal Novel Organic Cation Transporter 2-Targeting Oral Prodrugs

J Med Chem. 2017 Mar 23;60(6):2552-2561. doi: 10.1021/acs.jmedchem.7b00049. Epub 2017 Mar 7.

Abstract

Novel organic cation transporter 2 (OCTN2, SLC22A5) is responsible for the uptake of carnitine through the intestine and, therefore, might be a promising molecular target for designing oral prodrugs. Poor permeability and rapid metabolism have greatly restricted the oral absorption of gemcitabine. We here describe the design of intestinal OCTN2-targeting prodrugs of gemcitabine by covalent coupling of l-carnitine to its N4-amino group via different lipophilic linkages. Because of the high OCTN2 affinity, the hexane diacid-linked prodrug demonstrated significantly improved stability (3-fold), cellular permeability (15-fold), and oral bioavailability (5-fold), while causing no toxicity as compared to gemcitabine. In addition, OCTN2-targeting prodrugs can simultaneously improve the permeability, solubility, and metabolic stability of gemcitabine. In summary, we present the first evidence that OCTN2 can act as a new molecular target for oral prodrug delivery and, importantly, the linkage carbon chain length is a key factor in modifying the affinity of the substrate for OCTN2.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antimetabolites, Antineoplastic / chemistry*
  • Antimetabolites, Antineoplastic / metabolism
  • Antimetabolites, Antineoplastic / pharmacokinetics*
  • Caco-2 Cells
  • Carnitine / chemistry
  • Carnitine / metabolism
  • Carnitine / pharmacokinetics
  • Deoxycytidine / analogs & derivatives*
  • Deoxycytidine / chemistry
  • Deoxycytidine / metabolism
  • Deoxycytidine / pharmacokinetics
  • Gemcitabine
  • HEK293 Cells
  • Humans
  • Mice
  • Molecular Docking Simulation
  • Organic Cation Transport Proteins / metabolism*
  • Prodrugs / chemistry*
  • Prodrugs / metabolism
  • Prodrugs / pharmacokinetics*
  • Solute Carrier Family 22 Member 5
  • Tissue Distribution

Substances

  • Antimetabolites, Antineoplastic
  • Organic Cation Transport Proteins
  • Prodrugs
  • SLC22A5 protein, human
  • Solute Carrier Family 22 Member 5
  • Deoxycytidine
  • Carnitine
  • Gemcitabine