Graft-versus-leukemia effect of HLA-haploidentical central-memory T-cells expanded with leukemic APCs and modified with a suicide gene

Mol Ther. 2013 Feb;21(2):466-75. doi: 10.1038/mt.2012.227. Epub 2012 Nov 13.

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA)-haploidentical family donor (haplo-HSCT) is a readily available and potentially curative option for high-risk leukemia. In haplo-HSCT, alloreactivity plays a major role in the graft-versus-leukemia (GVL) effect, which, however, is frequently followed by relapse due to emerging leukemic cell variants that have lost the unshared HLA haplotype as a mechanism of immune escape. We report that stimulation of HLA-haploidentical donor T lymphocytes with leukemic antigen-presenting cells (L-APCs) expands a population of leukemia-reactive T cells, which, besides alloreactivity to unshared HLAs, contain leukemia-associated specificities restricted by shared HLAs. According to a preferential central-memory (T(CM)) phenotype and to high interleukin (IL)-7Rα expression, these T cells persist in vivo and sustain a major GVL effect in a clinically relevant xenograft model. Moreover, we demonstrate that modifying L-APC-expanded T cells to express the herpes simplex virus thymidine kinase (HSV-tk) suicide gene enables their elimination with the prodrug ganciclovir (GCV), therefore providing a safety switch in case of graft-versus-host disease (GVHD). These results warrant the clinical investigation of L-APC-expanded T cells modified with a suicide gene in the setting of haplo-HSCT.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Animals
  • Cell Line, Tumor
  • Disease Models, Animal
  • Flow Cytometry
  • Ganciclovir / pharmacology
  • Gene Expression Regulation, Neoplastic*
  • Genes, Transgenic, Suicide / genetics*
  • Genes, Transgenic, Suicide / immunology
  • Genes, Wilms Tumor
  • Genetic Therapy
  • Graft vs Host Disease / genetics
  • Graft vs Host Disease / immunology
  • Graft vs Host Disease / therapy
  • Graft vs Leukemia Effect / genetics*
  • HLA Antigens / genetics*
  • HLA Antigens / immunology
  • Hematopoietic Stem Cell Transplantation / methods
  • Humans
  • Leukemia / genetics*
  • Leukemia / pathology
  • Leukemia / therapy
  • Mice
  • Mice, SCID
  • Middle Aged
  • T-Lymphocytes / immunology*
  • T-Lymphocytes / transplantation
  • Young Adult

Substances

  • HLA Antigens
  • Ganciclovir