LILRB4 represents a promising target for immunotherapy by dual targeting tumor cells and myeloid-derived suppressive cells in multiple myeloma

Haematologica. 2024 Nov 1;109(11):3650-3669. doi: 10.3324/haematol.2024.285099.

Abstract

Multiple myeloma (MM) remains an incurable hematologic malignancy. Despite tremendous advances in the treatment of this disease, about 10% of patients still have very poor outcomes with a median overall survival of less than 24 months. Our study aimed to underscore the critical mechanisms pertaining to rapid disease progression and provide novel therapeutic choices for these ultrahigh-risk patients. We utilized single-cell transcriptomic sequencing to dissect the characteristic bone marrow niche of patients who survived less than 2 years (EM24). Notably, enrichment of a LILRB4high pre-mature plasma-cell cluster was observed in EM24 patients compared to patients with durable remission. This cluster exhibited aggressive proliferation and a drug-resistance phenotype. High levels of LILRB4 promoted MM clonogenicity and progression. Clinically, high expression of LILRB4 was correlated with poor prognosis in both newly diagnosed MM patients and relapsed/ refractory MM patients. ATAC-sequencing analysis identified that pronounced chromosomal accessibility caused the elevation of LILRB4 on MM cells. CRISPR-Cas9 deletion of LILRB4 alleviated the growth of MM cells, inhibited the immunosuppressive function of myeloid-derived suppressive cells (MDSC), and further rescued T-cell dysfunction in the MM microenvironment. Greater infiltration of MDSC was observed in EM24 patients. We therefore generated an innovative T-cell receptor-based chimeric antigen receptor T cell, LILRB4-STAR-T. Cytotoxicity experiments demonstrated that LILRB4-STAR-T cells efficaciously eliminated tumor cells and impeded MDSC function. In conclusion, our study elucidates that LILRB4 is an ideal biomarker and promising immunotherapy target for high-risk MM. LILRB4-STAR-T-cell immunotherapy is promising against both tumor cells and the immunosuppressive tumor microenvironment in MM.

MeSH terms

  • Animals
  • Biomarkers, Tumor
  • Cell Line, Tumor
  • Humans
  • Immunotherapy* / methods
  • Membrane Glycoproteins* / genetics
  • Membrane Glycoproteins* / immunology
  • Molecular Targeted Therapy
  • Multiple Myeloma* / genetics
  • Multiple Myeloma* / immunology
  • Multiple Myeloma* / mortality
  • Multiple Myeloma* / pathology
  • Multiple Myeloma* / therapy
  • Myeloid-Derived Suppressor Cells* / immunology
  • Myeloid-Derived Suppressor Cells* / metabolism
  • Prognosis
  • Receptors, Immunologic* / genetics
  • Receptors, Immunologic* / metabolism
  • Tumor Microenvironment / immunology

Substances

  • LILRB4 protein, human
  • Receptors, Immunologic
  • Membrane Glycoproteins
  • Biomarkers, Tumor

Grants and funding

Funding: This research was funded by the National Natural Science Foundation of China (82341211, 82370210, and 82170194) and the CAMS Innovation Fund for Medical Sciences (CIFMS 2022-I2M1-022 and CIFMS 2021-I2M1-040). The funder had no role in the study design, data collection, analysis, and interpretation, or in the writing of this manuscript.