High-throughput protein target mapping enables accelerated bioactivity discovery for ToxCast and PFAS compounds

Proc Natl Acad Sci U S A. 2026 Jun 23;123(25):e2515940123. doi: 10.1073/pnas.2515940123. Epub 2026 Jun 5.

Abstract

Chemical pollution is a global threat to human health, yet the toxicity mechanisms of most contaminants remains unknown. Here, we applied an ultrahigh-throughput affinity selection-mass spectrometry (AS-MS) platform to systematically identify protein targets of prioritized chemical contaminants. After benchmarking the platform, we screened 50 human proteins against 481 prioritized chemicals, including 446 ToxCast chemicals and 35 per- and polyfluoroalkyl substances (PFAS). Among 24,050 interactions assessed, we discovered 35 interactions involving 13 proteins, with fatty acid-binding proteins (FABPs) emerging as the most ligandable protein family. Given this, we selected FABPs for further validation, which revealed a distinct PFAS binding pattern: legacy PFAS selectively bound to FABP1, whereas replacement compounds, perfluoroether carboxylic acids, unexpectedly interacted with all FABPs. X-ray crystallography further revealed that the ether group enhances the molecular flexibility of alternative PFAS to accommodate the binding pockets of FABPs. Our findings demonstrate that AS-MS is a robust platform for the discovery of protein targets beyond the scope of ToxCast and highlight the broader protein-binding spectrum of alternative PFAS as potential regrettable substitutes.

Keywords: PFAS; ToxCast; protein–ligand interaction.

MeSH terms

  • Crystallography, X-Ray
  • Environmental Pollutants* / chemistry
  • Environmental Pollutants* / toxicity
  • Fatty Acid-Binding Proteins* / chemistry
  • Fatty Acid-Binding Proteins* / metabolism
  • Fluorocarbons* / chemistry
  • Fluorocarbons* / toxicity
  • High-Throughput Screening Assays* / methods
  • Humans
  • Ligands
  • Mass Spectrometry / methods
  • Protein Binding

Substances

  • Fluorocarbons
  • Fatty Acid-Binding Proteins
  • Ligands
  • Environmental Pollutants

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