Immunological defects after suicide gene therapy of experimental graft-versus-host disease

Hum Gene Ther. 1999 Nov 1;10(16):2701-7. doi: 10.1089/10430349950016744.

Abstract

Donor T cells are beneficial for engraftment, immune reconstitution, and antileukemic effects after allogeneic marrow transplantation, but they also cause graft-versus-host disease. Treatment with ganciclovir can control graft-versus-host disease if donor T cells are genetically engineered to express viral thymidine kinase. Clinical protocols with thymidine kinase-expressing T cells currently prescribe the curative use of ganciclovir for genetic immunosuppression only after clinical manifestations of graft-versus-host disease have appeared. The aim of this work was to compare early/preventive versus delayed/curative treatment of GVHD. Here, we found that ganciclovir administered early after experimental marrow transplantation was highly effective in preventing graft-versus-host disease caused by thymidine kinase-expressing T cells, and surviving recipient mice were able to mount a T cell-dependent B cell response. In contrast, curative ganciclovir administration later after transplantation was much less effective in treating graft-versus-host disease and surviving recipients had markedly impaired immune function. These findings should be considered in the development of future clinical trials using thymidine kinase-expressing T cells; to date, such trials have envisaged the use of GCV to treat only declared graft-versus-host disease. The use of thymidine kinase-expressing T cells for conditional elimination of activated T cells after allogeneic marrow transplantation offers a promising new approach for the control of graft-versus-host disease. The versatility of the thymidine kinase/ganciclovir system offers clinical options depending on whether thymidine kinase-expressing T cells are infused at the time of bone marrow transplantation or in a delayed manner, and depending on whether GCV is administered in an early/preventive or delayed/curative manner. The rationale underlying these options is more complex than it may appear and is likely to have a profound impact on the efficacy of such treatments. In the present work, we analyze the immunological impact when GCV is administered in an early/preventive or delayed/curative manner. Our results demonstrate that the delayed/curative strategy is clearly associated with severe immunological defects. To our knowledge, this is the first report of immunodeficiency subsequent to suicide gene therapy for GVHD.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Bone Marrow Transplantation
  • DNA / metabolism
  • Female
  • Ganciclovir / pharmacology*
  • Genetic Therapy / methods*
  • Graft vs Host Disease / immunology*
  • Graft vs Host Disease / mortality
  • Graft vs Host Disease / therapy*
  • Immunoglobulin G / blood
  • Immunoglobulin M / metabolism
  • Liver / pathology
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred Strains
  • Spleen / pathology
  • Survival Rate
  • Thymidine Kinase / drug effects
  • Thymidine Kinase / genetics

Substances

  • Immunoglobulin G
  • Immunoglobulin M
  • DNA
  • Thymidine Kinase
  • Ganciclovir