Targeting amyloid-β pathology by chimeric antigen receptor astrocyte (CAR-A) therapy

Science. 2026 Mar 5;391(6789):eads3972. doi: 10.1126/science.ads3972. Epub 2026 Mar 5.

Abstract

Alzheimer's disease (AD) is the leading cause of dementia and is characterized by progressive amyloid accumulation followed by tau-mediated neurodegeneration. Despite advances in anti-amyloid immunotherapies, important limitations remain, highlighting the need for new therapeutic strategies. Here, we introduce anti-amyloid chimeric antigen receptors expressed in astrocytes (CAR-A) and validate their function in vitro. We show that two CAR-A designs reduce amyloid and associated pathology after plaque formation and prevent early plaque deposition in vivo. Single-nucleus RNA sequencing shows that CAR-A treatment induces a distinct glial response to amyloid pathology involving coordinated activity of astrocytes and microglia. Each construct additionally elicits distinctive, receptor-specific effects in astrocytes or microglia. Together, these findings support the therapeutic potential of CAR-A as a disease-modifying strategy for AD.

MeSH terms

  • Alzheimer Disease* / pathology
  • Alzheimer Disease* / therapy
  • Amyloid beta-Peptides* / immunology
  • Amyloid beta-Peptides* / metabolism
  • Animals
  • Astrocytes* / immunology
  • Astrocytes* / metabolism
  • Humans
  • Immunotherapy, Adoptive* / methods
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Microglia / immunology
  • Microglia / metabolism
  • Plaque, Amyloid* / pathology
  • Plaque, Amyloid* / therapy
  • Receptors, Chimeric Antigen* / genetics
  • Receptors, Chimeric Antigen* / immunology

Substances

  • Amyloid beta-Peptides
  • Receptors, Chimeric Antigen