Gut microbiota-derived hexa-acylated lipopolysaccharides enhance cancer immunotherapy responses

Nat Microbiol. 2025 Mar;10(3):795-807. doi: 10.1038/s41564-025-01930-y. Epub 2025 Feb 10.

Abstract

The gut microbiome modulates immunotherapy treatment responses, and this may explain why immune checkpoint inhibitors, such as anti-PD-1, are only effective in some patients. Previous studies correlated lipopolysaccharide (LPS)-producing gut microbes with poorer prognosis; however, LPS from diverse bacterial species can range from immunostimulatory to inhibitory. Here, by functionally analysing faecal metagenomes from 112 patients with melanoma, we found that a subset of LPS-producing bacteria encoding immunostimulatory hexa-acylated LPS was enriched in microbiomes of clinical responders. In an implanted tumour mouse model of anti-PD-1 treatment, microbiota-derived hexa-acylated LPS was required for effective anti-tumour immune responses, and LPS-binding antibiotics and a small-molecule TLR4 antagonist abolished anti-PD-1 efficacy. Conversely, oral administration of hexa-acylated LPS to mice significantly augmented anti-PD-1-mediated anti-tumour immunity. Penta-acylated LPS did not improve anti-PD-1 efficacy in vivo and inhibited hexa-acylated LPS-induced immune activation in vitro. Microbiome hexa-acylated LPS therefore represents an accessible predictor and potential enhancer of immunotherapy responses.

MeSH terms

  • Acylation
  • Animals
  • Bacteria / classification
  • Bacteria / genetics
  • Bacteria / metabolism
  • Cell Line, Tumor
  • Feces / microbiology
  • Female
  • Gastrointestinal Microbiome* / drug effects
  • Gastrointestinal Microbiome* / immunology
  • Humans
  • Immune Checkpoint Inhibitors / pharmacology
  • Immune Checkpoint Inhibitors / therapeutic use
  • Immunotherapy* / methods
  • Lipopolysaccharides* / chemistry
  • Lipopolysaccharides* / immunology
  • Lipopolysaccharides* / metabolism
  • Lipopolysaccharides* / pharmacology
  • Male
  • Melanoma* / immunology
  • Melanoma* / microbiology
  • Melanoma* / therapy
  • Mice
  • Mice, Inbred C57BL
  • Programmed Cell Death 1 Receptor / antagonists & inhibitors
  • Programmed Cell Death 1 Receptor / immunology
  • Toll-Like Receptor 4 / antagonists & inhibitors

Substances

  • Lipopolysaccharides
  • Programmed Cell Death 1 Receptor
  • Immune Checkpoint Inhibitors
  • Toll-Like Receptor 4