Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes

In the past two decades, several tissues have been generated from the differentiation of human pluripotent stem cells (hPSCs) to model development or disease, and for use in drug testing and cell replacement therapies. A frontliner of hPSC-derived tissues used in cell replacement therapies are the p...

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Main Authors: Mohammed Usama, Ying Deng, Yiran Chen, Théa Milland, Mohan Malleshaiah, Yasaman Aghazadeh
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-08-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1625439/full
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author Mohammed Usama
Mohammed Usama
Ying Deng
Ying Deng
Yiran Chen
Théa Milland
Théa Milland
Mohan Malleshaiah
Mohan Malleshaiah
Mohan Malleshaiah
Yasaman Aghazadeh
Yasaman Aghazadeh
Yasaman Aghazadeh
author_facet Mohammed Usama
Mohammed Usama
Ying Deng
Ying Deng
Yiran Chen
Théa Milland
Théa Milland
Mohan Malleshaiah
Mohan Malleshaiah
Mohan Malleshaiah
Yasaman Aghazadeh
Yasaman Aghazadeh
Yasaman Aghazadeh
author_sort Mohammed Usama
collection DOAJ
description In the past two decades, several tissues have been generated from the differentiation of human pluripotent stem cells (hPSCs) to model development or disease, and for use in drug testing and cell replacement therapies. A frontliner of hPSC-derived tissues used in cell replacement therapies are the pancreatic cells, which have entered multiple clinical trials since 2014 for the treatment of type 1 diabetes (T1D). Despite challenges in early trials, the detection of endogenous C-peptide in recipients was encouraging. The results and challenges of these trials inspired new areas of research, leading to incremental advances in cell differentiation and delivery technologies, and a deeper understanding of the transplantation microenvironment to enhance therapeutic efficacy and longevity. Reports from the most recent trials demonstrated success in reducing or eliminating exogenous insulin administration for people with T1D, increasing hope for a cure for T1D via regenerative medicine. Recent efforts can be broadly categorized into: (1) improving the cell product as surrogates of native beta cells, (2) promoting engraftment post-transplant to support cell survival, integration into the host, and endocrine function, and (3) developing immunomodulation strategies to reduce or circumvent immunosuppression regimen. In this review, we discuss recent and emerging advances in these three areas and the potential, risk, and scalability of experimental models to the clinic.
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institution Kabale University
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spelling doaj-art-1bce72497a6d48dc8e05eb06a1870b0c2025-08-20T03:57:39ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-08-011610.3389/fimmu.2025.16254391625439Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetesMohammed Usama0Mohammed Usama1Ying Deng2Ying Deng3Yiran Chen4Théa Milland5Théa Milland6Mohan Malleshaiah7Mohan Malleshaiah8Mohan Malleshaiah9Yasaman Aghazadeh10Yasaman Aghazadeh11Yasaman Aghazadeh12Institute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaDivision of Clinical and Translational Research, Department of Medicine, McGill University, Montréal, QC, CanadaInstitute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaDepartment of Medicine, University of Montréal, Montréal, QC, CanadaInstitute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaInstitute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaDepartment of Anatomy and Cell Biology, McGill University, Montréal, QC, CanadaInstitute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaDivision of Clinical and Translational Research, Department of Medicine, McGill University, Montréal, QC, CanadaDepartment of Biochemistry and Molecular Medicine, University of Montréal, Montréal, QC, CanadaInstitute de Recherches Cliniques de Montréal (IRCM), Montréal, QC, CanadaDivision of Clinical and Translational Research, Department of Medicine, McGill University, Montréal, QC, CanadaDepartment of Medicine, University of Montréal, Montréal, QC, CanadaIn the past two decades, several tissues have been generated from the differentiation of human pluripotent stem cells (hPSCs) to model development or disease, and for use in drug testing and cell replacement therapies. A frontliner of hPSC-derived tissues used in cell replacement therapies are the pancreatic cells, which have entered multiple clinical trials since 2014 for the treatment of type 1 diabetes (T1D). Despite challenges in early trials, the detection of endogenous C-peptide in recipients was encouraging. The results and challenges of these trials inspired new areas of research, leading to incremental advances in cell differentiation and delivery technologies, and a deeper understanding of the transplantation microenvironment to enhance therapeutic efficacy and longevity. Reports from the most recent trials demonstrated success in reducing or eliminating exogenous insulin administration for people with T1D, increasing hope for a cure for T1D via regenerative medicine. Recent efforts can be broadly categorized into: (1) improving the cell product as surrogates of native beta cells, (2) promoting engraftment post-transplant to support cell survival, integration into the host, and endocrine function, and (3) developing immunomodulation strategies to reduce or circumvent immunosuppression regimen. In this review, we discuss recent and emerging advances in these three areas and the potential, risk, and scalability of experimental models to the clinic.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1625439/fullbeta cellsisletstransplantationhuman pluripotent stem cellsvascularizationimmune cells
spellingShingle Mohammed Usama
Mohammed Usama
Ying Deng
Ying Deng
Yiran Chen
Théa Milland
Théa Milland
Mohan Malleshaiah
Mohan Malleshaiah
Mohan Malleshaiah
Yasaman Aghazadeh
Yasaman Aghazadeh
Yasaman Aghazadeh
Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
Frontiers in Immunology
beta cells
islets
transplantation
human pluripotent stem cells
vascularization
immune cells
title Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
title_full Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
title_fullStr Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
title_full_unstemmed Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
title_short Navigating challenges in human pluripotent stem cell-derived islet therapy for type 1 diabetes
title_sort navigating challenges in human pluripotent stem cell derived islet therapy for type 1 diabetes
topic beta cells
islets
transplantation
human pluripotent stem cells
vascularization
immune cells
url https://www.frontiersin.org/articles/10.3389/fimmu.2025.1625439/full
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