Cited by 216Pancreatic function and diabetesPluripotent Stem Cells ResearchDiabetes and associated disorders

The DOI matches its Crossref record; 188 of the 191 references it cites were found in a registry or an index; 235 of the 317 sources its claims cite were checked for support.

Developments in stem cell-derived islet replacement therapy for treating type 1 diabetes

Open access

Abstract

The generation of islet-like endocrine clusters from human pluripotent stem cells (hPSCs) has the potential to provide an unlimited source of insulin-producing β cells for the treatment of diabetes. In order for this cell therapy to become widely adopted, highly functional and well-characterized stem cell-derived islets (SC-islets) need to be manufactured at scale. Furthermore, successful SC-islet replacement strategies should prevent significant cell loss immediately following transplantation and avoid long-term immune rejection. This review highlights the most recent advances in the generation and characterization of highly functional SC-islets as well as strategies to ensure graft viability and safety after transplantation.

Overview

AI snapshot

Written from the paper’s full text.

TL;DR

This review summarizes advances in stem cell-derived islet (SC-islet) replacement therapy for type 1 diabetes, covering differentiation protocols, single-cell characterization, immune protection, and clinical translation. It argues SC-islets can improve glycemic control but still face functional immaturity, off-target cells, immune rejection, post-transplant stress, and manufacturing/safety hurdles.

Key findings

  • Clinical islet transplantation established feasibility: the Edmonton Protocol led to insulin independence in all seven patients at 1 year, though all but one required supplemental insulin by 10 years; later 5-year insulin independence rates reached 50–80%, and immediate graft loss was reduced from ≥50% to closer to 25%.
  • SC-islet clinical trials show promise but limitations: ViaCyte’s PEC-Direct produced glucose-responsive C-peptide 6–9 months post-transplant, but levels were too low for measurable clinical benefit; Vertex’s VX-880 preliminary press-release results improved glycemic control in two T1D patients, with slower effects than in rodents.
  • Single-cell studies reveal heterogeneity: scRNA-seq of 40,444 and 87,769 cells identified off-target SC-EC cells (TPH1/LMX1A, serotonin) sharing a NEUROG3+ progenitor with SC-β cells; multiomic analyses found >600 genes, ~350 promoter regions, and ~250 transcription factor binding motifs increased after 6 months in vivo.
  • Manufacturing/dose challenges remain: 2–5 × 10^6 SC-islet cells reverse hyperglycemia in mice, but humans may need ~10^9 cells per patient, so 1,000 patients require ~10^12 cells; the largest reported SC-islet differentiation was in 500 mL spinner flasks. Encapsulation with triazole–thiomorpholine dioxide alginate restored glycemic control in mice for at least 174 days without fibrosis.

Methods

  • Narrative review; no primary experiments or systematic search described.
  • Synthesizes preclinical rodent and non-human primate studies, human clinical trials (ViaCyte NCT02239354/NCT03163511; Vertex NCT04786262), and single-cell RNA-seq/ATAC-seq/multiomic studies.
  • Main analysis is qualitative synthesis of advances and challenges in SC-islet generation, immune protection, transplantation, scale-up, and safety.

Limitations

  • Stated: current SC-β cells remain less functional than primary human islets, transcriptionally/chromatin immature, and differentiation produces off-target SC-EC cells; immune rejection/immunosuppression, immediate post-transplant cell loss, scale-up/cryopreservation, and long-term safety remain unresolved.
  • Evident: as a narrative review, it does not systematically assess evidence quality; some cited clinical findings are preliminary, from a press release, or preprints under peer review.

Model-written summary. Check it against the paper before relying on it.

Integrity signals

Registry

Crossref · 10.1016/j.stem.2023.04.002

Cites

188 of 188 references verified0 citation errors · 0 not checkable

Statistics

Nothing to recompute.

Verification

Claims the paper makes

81 Supported · 65 Partly supported · 46 Own interpretation · 22 No full text of the sources · 16 No basis given · 12 Supported so far · 9 Not checked yet · 2 Couldn’t confirm · 2 Sources not found · 1 Not supported · accuracy not yet measured

Verify all 14 references — free account

Show all 166 claims

  • It may state two separate claims as one. 76
  • Probably not a substantial claim. 16
  • The quote only partly states this claim. 3
90 of 90
  • The paper relies on this without support.

    The number of donor pancreases available for islet isolation is limited.

    “Not only are the number of pancreases available for islet isolation limited, but transplanting cells from multiple genetic backgrounds per patient could exacerbate immune rejection and graft failure.25,26”
  • The paper relies on this without support.

    Many patients with T1D still fail to achieve their target blood glucose goals despite advances in insulin pumps and continuous glucose monitors.

    “Despite these advances, however, many patients still fail to achieve their target goals.”
  • The paper relies on this without support.

    Life-long immunosuppression has potentially severe adverse side effects.

    “the potentially severe adverse side effects of life-long immunosuppression may not outweigh the benefits of further improved glycemic control.”
  • The paper relies on this without support.

    Transplanted SC-islets are targeted by the host immune system as foreign, resulting in graft rejection.

    “As with the transplantation of any allogenic material, transplanted SC-islets are targeted by the host immune system as foreign, resulting in graft rejection.”
  • The paper relies on this without support.

    Type 1 diabetes itself is an autoimmune disease that specifically targets β cells.

    “An additional complication is that T1D itself is an autoimmune disease that specifically targets β cells.”
  • The paper relies on this without support.

    Deleting HLAs from cells causes them to be targeted by cells of the innate immune system, such as natural killer (NK) cells and macrophages.

    “Deleting HLAs from cells, however, causes them to be targeted by cells of the innate immune system, such as natural killer (NK) cells and macrophages.”
  • The paper relies on this without support.

    Due to the high metabolic demands of insulin production and secretion, β cells are particularly susceptible to stress.

    “Due to the high metabolic demands of insulin production and secretion, β cells are particularly susceptible to stress.”
  • The paper relies on this without support.

    Native islets are highly vascularized within the pancreas to receive the proper nutrients and high oxygen supply required for the production and secretion of large amounts of insulin.

    “Native islets are highly vascularized within the pancreas to receive the proper nutrients and high oxygen supply required for the production and secretion of large amounts of insulin.”
  • The authors state this as an assumption.

    The required dose of SC-islets for humans is similar to the dose of human islets from deceased donors.

    “Assuming a similar dose as with human islets from deceased donors,175,176 on the order of 109 cells will be required per patient.”
  • The paper relies on this without support.

    Large-scale bioreactors have not yet been extended to hPSCs.

    “To-date, however, such large scales have not been extended to hPSCs”
  • The paper relies on this without support.

    All well-established protocols require approximately one month of culture to achieve functional SC-islets, with additional time needed for SC-β cell maturation.

    “All well-established protocols require approximately one month of culture to achieve functional SC-islets, with additional time needed for SC-β cell maturation.”
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Metadata

Authors
Hogrebe, N. J., Ishahak, M., & Millman, J. R.
Affiliations
Washington University in St. Louis (US)
Corresponding author
  • Hogrebe, Nathaniel J.
  • Millman, Jeffrey R.
Venue
Cell Stem Cell
Publisher
Elsevier BV
Published
2023-05
Volume
30
Issue
5
Pages
530-548
ISSN
  • 1875-9777 (ISSN-L)
  • 1934-5909
OpenAlex
W4372335371
Cited by
210
References
191
Citations by year
2026: 59 · 2025: 75 · 2024: 61 · 2023: 15
FWCI
14.00
Type
journal article
Language
en
Open access
Locations
  • Cell stem cell (publishedVersion, cc-by-nc-nd)
  • PubMed (publishedVersion)
  • PubMed Central (acceptedVersion)
  • Digital Commons@Becker (Washington University School of Medicine) (submittedVersion, cc-by-nc-nd)
Funders
  • JDRF (3-SRA-2023-1295-S-B, 1-FAC-2023-1316-A-N)
  • Washington University in St Louis
  • NIH (T32DK007120, R01DK127497)
  • Edward Mallinckrodt, Jr. Foundation
Topics
  • Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Pancreatic function and diabetes
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Diabetes and associated disorders
Keywords
stem cell-derived islets, type 1 diabetes, human pluripotent stem cells, beta cell differentiation, cell therapy, graft viability, immune rejection, transplantation
MeSH terms
Cell Differentiation, Diabetes Mellitus, Type 1, Humans, Islets of Langerhans, Pluripotent Stem Cells, Insulin-Secreting Cells

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