Therapeutic bispecific T-cell engager antibody targeting the intracellular oncoprotein WT1

Nat Biotechnol. 2015 Oct;33(10):1079-86. doi: 10.1038/nbt.3349. Epub 2015 Sep 21.

Abstract

Intracellular tumor antigens presented on the cell surface in the context of human leukocyte antigen (HLA) molecules have been targeted by T cell-based therapies, but there has been little progress in developing small-molecule drugs or antibodies directed to these antigens. Here we describe a bispecific T-cell engager (BiTE) antibody derived from a T-cell receptor (TCR)-mimic monoclonal antibody (mAb) ESK1, which binds a peptide derived from the intracellular oncoprotein WT1 presented on HLA-A*02:01. Despite the very low density of the complexes at the cell surface, ESK1-BiTE selectively activated and induced proliferation of cytolytic human T cells that killed cells from multiple leukemias and solid tumors in vitro and in mice. We also discovered that in an autologous in vitro setting, ESK1-BiTE induced a robust secondary CD8 T-cell response specific for tumor-associated antigens other than WT1. Our study provides an approach that targets tumor-specific intracellular antigens without using cell therapy and suggests that epitope spreading could contribute to the therapeutic efficacy of this BiTE.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibodies, Monoclonal / immunology
  • Antibodies, Monoclonal / therapeutic use*
  • Antigens, Neoplasm / immunology
  • Cancer Vaccines / immunology
  • Cancer Vaccines / therapeutic use
  • Cell Line, Tumor
  • Humans
  • Mice
  • Molecular Targeted Therapy / methods
  • Neoplasms, Experimental / immunology*
  • Neoplasms, Experimental / therapy*
  • Oncogene Proteins / immunology
  • Repressor Proteins / immunology*
  • T-Lymphocytes / immunology*
  • Treatment Outcome
  • WT1 Proteins

Substances

  • Antibodies, Monoclonal
  • Antigens, Neoplasm
  • Cancer Vaccines
  • Oncogene Proteins
  • Repressor Proteins
  • WT1 Proteins
  • WT1 protein, mouse