The metabolite BH4 controls T cell proliferation in autoimmunity and cancer

Nature. 2018 Nov;563(7732):564-568. doi: 10.1038/s41586-018-0701-2. Epub 2018 Nov 7.

Abstract

Genetic regulators and environmental stimuli modulate T cell activation in autoimmunity and cancer. The enzyme co-factor tetrahydrobiopterin (BH4) is involved in the production of monoamine neurotransmitters, the generation of nitric oxide, and pain1,2. Here we uncover a link between these processes, identifying a fundamental role for BH4 in T cell biology. We find that genetic inactivation of GTP cyclohydrolase 1 (GCH1, the rate-limiting enzyme in the synthesis of BH4) and inhibition of sepiapterin reductase (the terminal enzyme in the synthetic pathway for BH4) severely impair the proliferation of mature mouse and human T cells. BH4 production in activated T cells is linked to alterations in iron metabolism and mitochondrial bioenergetics. In vivo blockade of BH4 synthesis abrogates T-cell-mediated autoimmunity and allergic inflammation, and enhancing BH4 levels through GCH1 overexpression augments responses by CD4- and CD8-expressing T cells, increasing their antitumour activity in vivo. Administration of BH4 to mice markedly reduces tumour growth and expands the population of intratumoral effector T cells. Kynurenine-a tryptophan metabolite that blocks antitumour immunity-inhibits T cell proliferation in a manner that can be rescued by BH4. Finally, we report the development of a potent SPR antagonist for possible clinical use. Our data uncover GCH1, SPR and their downstream metabolite BH4 as critical regulators of T cell biology that can be readily manipulated to either block autoimmunity or enhance anticancer immunity.

Publication types

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

MeSH terms

  • Administration, Oral
  • Alcohol Oxidoreductases / antagonists & inhibitors
  • Alcohol Oxidoreductases / metabolism
  • Animals
  • Autoimmune Diseases / drug therapy
  • Autoimmune Diseases / immunology*
  • Autoimmune Diseases / pathology
  • Biopterins / analogs & derivatives*
  • Biopterins / biosynthesis
  • Biopterins / metabolism
  • Biopterins / pharmacology
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Coenzymes / metabolism
  • Enzyme Inhibitors / administration & dosage
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • Female
  • GTP Cyclohydrolase / genetics
  • GTP Cyclohydrolase / metabolism
  • Humans
  • Hypersensitivity / immunology
  • Iron / metabolism
  • Kynurenine / metabolism
  • Kynurenine / pharmacology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mitochondria / metabolism
  • Neoplasms / drug therapy
  • Neoplasms / immunology*
  • Neoplasms / pathology
  • T-Lymphocytes / cytology*
  • T-Lymphocytes / drug effects
  • T-Lymphocytes / immunology*
  • T-Lymphocytes / metabolism

Substances

  • Coenzymes
  • Enzyme Inhibitors
  • Biopterins
  • Kynurenine
  • Iron
  • Alcohol Oxidoreductases
  • sepiapterin reductase
  • GTP Cyclohydrolase
  • sapropterin