TNFR1 inhibition with a Nanobody protects against EAE development in mice

Sci Rep. 2017 Oct 20;7(1):13646. doi: 10.1038/s41598-017-13984-y.

Abstract

TNF has as detrimental role in multiple sclerosis (MS), however, anti-TNF medication is not working. Selective TNF/TNFR1 inhibition whilst sparing TNFR2 signaling reduces the pro-inflammatory effects of TNF but preserves the important neuroprotective signals via TNFR2. We previously reported the generation of a Nanobody-based selective inhibitor of human TNFR1, TROS that will be tested in experimental autoimmune encephalomyelitis (EAE). We specifically antagonized TNF/TNFR1 signaling using TROS in a murine model of MS, namely MOG35-55-induced EAE. Because TROS does not cross-react with mouse TNFR1, we generated mice expressing human TNFR1 in a mouse TNFR1-knockout background (hTNFR1 Tg), and we determined biodistribution of 99mTc-TROS and effectiveness of TROS in EAE in those mice. Biodistribution analysis demonstrated that intraperitoneally injected TROS is retained more in organs of hTNFR1 Tg mice compared to wild type mice. TROS was also detected in the cerebrospinal fluid (CSF) of hTNFR1 Tg mice. Prophylactic TROS administration significantly delayed disease onset and ameliorated its symptoms. Moreover, treatment initiated early after disease onset prevented further disease development. TROS reduced spinal cord inflammation and neuroinflammation, and preserved myelin and neurons. Collectively, our data illustrate that TNFR1 is a promising therapeutic target in MS.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Encephalomyelitis, Autoimmune, Experimental / pathology
  • Encephalomyelitis, Autoimmune, Experimental / prevention & control*
  • Humans
  • Immunologic Factors / pharmacokinetics
  • Immunologic Factors / pharmacology*
  • Male
  • Mice, Transgenic
  • Myelin-Oligodendrocyte Glycoprotein
  • Neuroprotective Agents / pharmacokinetics
  • Neuroprotective Agents / pharmacology*
  • Peptide Fragments
  • Receptors, Tumor Necrosis Factor, Type I / antagonists & inhibitors*
  • Receptors, Tumor Necrosis Factor, Type I / deficiency
  • Receptors, Tumor Necrosis Factor, Type I / genetics
  • Single-Domain Antibodies / pharmacology*
  • Spinal Cord / drug effects
  • Spinal Cord / metabolism
  • Spinal Cord / pathology
  • Technetium
  • Tumor Necrosis Factor-alpha / metabolism
  • Whole Body Imaging

Substances

  • Immunologic Factors
  • Myelin-Oligodendrocyte Glycoprotein
  • Neuroprotective Agents
  • Peptide Fragments
  • Receptors, Tumor Necrosis Factor, Type I
  • Single-Domain Antibodies
  • Technetium-99
  • Tnfrsf1a protein, mouse
  • Tumor Necrosis Factor-alpha
  • myelin oligodendrocyte glycoprotein (35-55)
  • Technetium