Drug-induced phospholipidosis confounds drug repurposing for SARS-CoV-2

Science. 2021 Jul 30;373(6554):541-547. doi: 10.1126/science.abi4708. Epub 2021 Jun 22.

Abstract

Repurposing drugs as treatments for COVID-19, the disease caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has drawn much attention. Beginning with sigma receptor ligands and expanding to other drugs from screening in the field, we became concerned that phospholipidosis was a shared mechanism underlying the antiviral activity of many repurposed drugs. For all of the 23 cationic amphiphilic drugs we tested, including hydroxychloroquine, azithromycin, amiodarone, and four others already in clinical trials, phospholipidosis was monotonically correlated with antiviral efficacy. Conversely, drugs active against the same targets that did not induce phospholipidosis were not antiviral. Phospholipidosis depends on the physicochemical properties of drugs and does not reflect specific target-based activities-rather, it may be considered a toxic confound in early drug discovery. Early detection of phospholipidosis could eliminate these artifacts, enabling a focus on molecules with therapeutic potential.

Publication types

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

MeSH terms

  • A549 Cells
  • Animals
  • Antiviral Agents / chemistry
  • Antiviral Agents / pharmacology*
  • Antiviral Agents / therapeutic use
  • Antiviral Agents / toxicity
  • COVID-19 / virology
  • COVID-19 Drug Treatment*
  • Cations
  • Chlorocebus aethiops
  • Dose-Response Relationship, Drug
  • Drug Repositioning*
  • Female
  • Humans
  • Lipidoses / chemically induced*
  • Mice
  • Microbial Sensitivity Tests
  • Phospholipids / metabolism*
  • SARS-CoV-2 / drug effects*
  • SARS-CoV-2 / physiology
  • Surface-Active Agents / chemistry
  • Surface-Active Agents / pharmacology
  • Surface-Active Agents / toxicity
  • Vero Cells
  • Virus Replication / drug effects

Substances

  • Antiviral Agents
  • Cations
  • Phospholipids
  • Surface-Active Agents