Systematically optimized BCMA/CS1 bispecific CAR-T cells robustly control heterogeneous multiple myeloma

Nat Commun. 2020 May 8;11(1):2283. doi: 10.1038/s41467-020-16160-5.

Abstract

Chimeric antigen receptor (CAR)-T cell therapy has shown remarkable clinical efficacy against B-cell malignancies, yet marked vulnerability to antigen escape and tumor relapse exists. Here we report the rational design and optimization of bispecific CAR-T cells with robust activity against heterogeneous multiple myeloma (MM) that is resistant to conventional CAR-T cell therapy targeting B-cell maturation antigen (BCMA). We demonstrate that BCMA/CS1 bispecific CAR-T cells exhibit superior CAR expression and function compared to T cells that co-express individual BCMA and CS1 CARs. Combination therapy with anti-PD-1 antibody further accelerates the rate of initial tumor clearance in vivo, while CAR-T cell treatment alone achieves durable tumor-free survival even upon tumor re-challenge. Taken together, the BCMA/CS1 bispecific CAR presents a promising treatment approach to prevent antigen escape in CAR-T cell therapy against MM, and the vertically integrated optimization process can be used to develop robust cell-based therapy against novel disease targets.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antigens, Neoplasm / immunology
  • B-Cell Maturation Antigen / metabolism
  • Cytotoxicity, Immunologic
  • Gene Editing
  • Humans
  • Immunologic Memory
  • Immunotherapy, Adoptive*
  • K562 Cells
  • Multiple Myeloma / immunology*
  • Multiple Myeloma / therapy*
  • Programmed Cell Death 1 Receptor / metabolism
  • Receptors, Chimeric Antigen / metabolism*
  • T-Lymphocytes / immunology

Substances

  • Antigens, Neoplasm
  • B-Cell Maturation Antigen
  • PDCD1 protein, human
  • Programmed Cell Death 1 Receptor
  • Receptors, Chimeric Antigen