Immune-evasive human islet-like organoids ameliorate diabetes

Nature. 2020 Oct;586(7830):606-611. doi: 10.1038/s41586-020-2631-z. Epub 2020 Aug 19.

Abstract

Islets derived from stem cells hold promise as a therapy for insulin-dependent diabetes, but there remain challenges towards achieving this goal1-6. Here we generate human islet-like organoids (HILOs) from induced pluripotent stem cells and show that non-canonical WNT4 signalling drives the metabolic maturation necessary for robust ex vivo glucose-stimulated insulin secretion. These functionally mature HILOs contain endocrine-like cell types that, upon transplantation, rapidly re-establish glucose homeostasis in diabetic NOD/SCID mice. Overexpression of the immune checkpoint protein programmed death-ligand 1 (PD-L1) protected HILO xenografts such that they were able to restore glucose homeostasis in immune-competent diabetic mice for 50 days. Furthermore, ex vivo stimulation with interferon-γ induced endogenous PD-L1 expression and restricted T cell activation and graft rejection. The generation of glucose-responsive islet-like organoids that are able to avoid immune detection provides a promising alternative to cadaveric and device-dependent therapies in the treatment of diabetes.

Publication types

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

MeSH terms

  • Animals
  • B7-H1 Antigen / genetics
  • B7-H1 Antigen / metabolism
  • Cell Line
  • Diabetes Mellitus, Experimental / immunology*
  • Diabetes Mellitus, Experimental / pathology*
  • Epigenesis, Genetic
  • Female
  • Glucose / metabolism
  • Graft Rejection
  • Heterografts
  • Homeostasis
  • Humans
  • Immune Evasion*
  • Immune Tolerance
  • Insulin Secretion
  • Islets of Langerhans / cytology*
  • Islets of Langerhans / immunology*
  • Islets of Langerhans Transplantation
  • Lymphocyte Activation
  • Male
  • Mice
  • Mice, Inbred NOD
  • Mice, SCID
  • Organoids / cytology*
  • Organoids / immunology*
  • Organoids / transplantation
  • T-Lymphocytes / cytology
  • T-Lymphocytes / immunology
  • Wnt Signaling Pathway / drug effects
  • Wnt4 Protein / metabolism
  • Wnt4 Protein / pharmacology

Substances

  • B7-H1 Antigen
  • CD274 protein, human
  • WNT4 protein, human
  • Wnt4 Protein
  • Glucose