TGN1412 Induces Lymphopenia and Human Cytokine Release in a Humanized Mouse Model

PLoS One. 2016 Mar 9;11(3):e0149093. doi: 10.1371/journal.pone.0149093. eCollection 2016.

Abstract

Therapeutic monoclonal antibodies (mAbs) such as the superagonistic, CD28-specific antibody TGN1412, or OKT3, an anti-CD3 mAb, can cause severe adverse events including cytokine release syndrome. A predictive model for mAb-mediated adverse effects, for which no previous knowledge on severe adverse events to be expected or on molecular mechanisms underlying is prerequisite, is not available yet. We used a humanized mouse model of human peripheral blood mononuclear cell-reconstituted NOD-RAG1-/-Aβ-/-HLADQ(tg+ or tg-)IL-2Rγc-/- mice to evaluate its predictive value for preclinical testing of mAbs. 2-6 hours after TGN1412 treatment, mice showed a loss of human CD45+ cells from the peripheral blood and loss of only human T cells after OKT3 injection, reminiscent of effects observed in mAb-treated humans. Moreover, upon OKT3 injection we detected selective CD3 downmodulation on T cells, a typical effect of OKT3. Importantly, we detected release of human cytokines in humanized mice upon both OKT3 and TGN1412 application. Finally, humanized mice showed severe signs of illness, a rapid drop of body temperature, and succumbed to antibody application 2-6 hours after administration. Hence, the humanized mouse model used here reproduces several effects and adverse events induced in humans upon application of the therapeutic mAbs OKT3 and TGN1412.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antibodies, Monoclonal, Humanized / adverse effects*
  • Antigens, CD / metabolism
  • Cytokines / blood*
  • Humans
  • Immunomodulation / drug effects
  • Lymphopenia / blood*
  • Lymphopenia / chemically induced*
  • Mice
  • Models, Animal
  • Muromonab-CD3 / pharmacology
  • T-Lymphocytes / immunology

Substances

  • Antibodies, Monoclonal, Humanized
  • Antigens, CD
  • Cytokines
  • Muromonab-CD3
  • TGN-1412

Grants and funding

The authors received no specific funding for this work. Funding of the work was obtained from the Paul-Ehrlich-Institut only.