Insulin expression and C-peptide in type 1 diabetes subjects implanted with stem cell-derived pancreatic endoderm cells in an encapsulation device

A. M.James Shapiro, David Thompson, Thomas W. Donner, Melena D. Bellin, Willa Hsueh, Jeremy Pettus, Jon Wilensky, Mark Daniels, Richard M. Wang, Eugene P. Brandon, Manasi S. Jaiman, Evert J. Kroon, Kevin A. D'Amour, Howard L. Foyt

Research output: Contribution to journalArticlepeer-review

135 Scopus citations

Abstract

These preliminary data from an ongoing first-in-human phase 1/2, open-label study provide proof-of-concept that pluripotent stem cell-derived pancreatic endoderm cells (PEC-01) engrafted in type 1 diabetes patients become islet cells releasing insulin in a physiologically regulated fashion. In this study of 17 subjects aged 22-57 with type 1 diabetes, PEC-01 cells were implanted subcutaneously in VC-02 macroencapsulation devices, allowing for direct vascularization of the cells. Engraftment and insulin expression were observed in 63% of VC-02 units explanted from subjects at 3–12 months post-implant. Six of 17 subjects (35.3%) demonstrated positive C-peptide as early as 6 months post-implant. Most reported adverse events were related to surgical implant or explant procedures (27.9%) or to side-effects of immunosuppression (33.7%). Initial data suggest that pluripotent stem cells, which can be propagated to the desired biomass and differentiated into pancreatic islet-like tissue, may offer a scalable, renewable alternative to pancreatic islet transplants.

Original languageEnglish (US)
Article number100466
JournalCell Reports Medicine
Volume2
Issue number12
DOIs
StatePublished - Dec 21 2021

Bibliographical note

Funding Information:
This work was supported in part by grants from CIRM (CLIN2-09672), JDRF (IDDP), and the Stem Cell Network of Canada. The authors thank Khaled Dajani, Peter A Senior, Kathleen Dungan, and Shumei Meng for their hard work on the trial.

Publisher Copyright:
© 2021 The Authors

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