Evaluation of single and combination therapies with tenofovir disoproxil fumarate and emtricitabine in vitro and in a robust mouse model supporting high levels of hepatitis B virus replication

Antimicrob Agents Chemother. 2012 Dec;56(12):6186-91. doi: 10.1128/AAC.01483-12. Epub 2012 Sep 17.

Abstract

Next-generation therapies for chronic hepatitis B virus (HBV) infection will involve combinations of established and/or experimental drugs. The current study investigated the in vitro and in vivo efficacy of tenofovir disoproxil fumarate (TDF) and/or emtricitabine [(-)-FTC] alone and in combination therapy for HBV infection utilizing the HepAD38 system (human hepatoblastoma cells transfected with HBV). Cellular pharmacology studies demonstrated increased levels of (-)-FTC triphosphate with coincubation of increasing concentrations of TDF, while (-)-FTC had no effect on intracellular tenofovir (TFV) diphosphate levels. Quantification of extracellular HBV by real-time PCR from hepatocytes demonstrated the anti-HBV activity with TDF, (-)-FTC, and their combination. Combination of (-)-FTC with TDF or TFV (ratio, 1:1) had a weighted average combination index of 0.7 for both combination sets, indicating synergistic antiviral effects. No cytotoxic effects were observed with any regimens. Using an in vivo murine model which develops robust HBV viremia in nude mice subcutaneously injected with HepAD38 cells, TDF (33 to 300 mg/kg of body weight/day) suppressed virus replication for up to 10 days posttreatment. At 300 mg/kg/day, (-)-FTC strongly suppressed virus titers to up to 14 days posttreatment. Combination therapy (33 mg/kg/day each drug) sustained suppression of virus titer/ml serum (<1 log(10) unit from pretreatment levels) at 14 days posttreatment, while single-drug treatments yielded virus titers 1.5 to 2 log units above the initial virus titers. There was no difference in mean alanine aminotransferase values or mean wet tumor weights for any of the groups, suggesting a lack of drug toxicity. TDF-(-)-FTC combination therapy provides more effective HBV suppression than therapy with each drug alone.

Publication types

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

MeSH terms

  • Adenine / analogs & derivatives*
  • Adenine / pharmacology
  • Adenine / therapeutic use
  • Adenine / toxicity
  • Animals
  • Antiviral Agents / pharmacology*
  • Antiviral Agents / therapeutic use*
  • Antiviral Agents / toxicity
  • Cell Line
  • Deoxycytidine / analogs & derivatives*
  • Deoxycytidine / pharmacology
  • Deoxycytidine / therapeutic use
  • Deoxycytidine / toxicity
  • Drug Combinations
  • Drug Interactions
  • Emtricitabine
  • Hepatitis B / drug therapy*
  • Hepatitis B / virology
  • Hepatitis B virus / drug effects*
  • Mice
  • Mice, Nude
  • Organophosphonates / pharmacology*
  • Organophosphonates / therapeutic use*
  • Organophosphonates / toxicity
  • Phosphorylation
  • Polymerase Chain Reaction
  • Reverse Transcriptase Inhibitors / pharmacology*
  • Reverse Transcriptase Inhibitors / therapeutic use
  • Reverse Transcriptase Inhibitors / toxicity
  • Tenofovir
  • Viremia / drug therapy
  • Viremia / virology
  • Virus Replication / drug effects*

Substances

  • Antiviral Agents
  • Drug Combinations
  • Organophosphonates
  • Reverse Transcriptase Inhibitors
  • Deoxycytidine
  • Tenofovir
  • Emtricitabine
  • Adenine