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Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes
Trevor Reichman, James F. Markmann, Jon S. Odorico, Piotr Witkowski, John J. Fung, Martin N. Wijkstrom, Fouad F Kandeel, Eelco J.P. de Koning et al.. New England Journal of Medicine, 2025-06-20
Topic: Brain proteins, microglia and lab models
A total of 14 participants (2 in part A and 12 in parts B and C) completed at least 12 months of follow-up and were included in the analyses. C-peptide was undetectable at baseline in all 14 participants. After zimislecel infusion, all the participants had engraftment and islet function, as evidenced by the detection of C-peptide. Neutropenia was the most common serious adverse event, occurring in 3 participants. Two deaths occurred - one caused by cryptococcal meningitis and one by severe dementia with agitation owing to the progression of preexisting neurocognitive impairment. All 12 participants in parts B and C were free of severe hypoglycemic events and had a glycated hemoglobin level of less than 7%; these participants spent more than 70% of the time in the target glucose range (70 to 180 mg per deciliter). Ten of the 12 participants (83%) had insulin independence and were not using exogenous insulin at day 365. The results of this small, short-term study involving persons with type 1 diabetes support the hypothesis that zimislecel can restore physiologic islet function, warranting further clinical investigation. (Funded by Vertex Pharmaceuticals; VX-880-101 FORWARD ClinicalTrials.gov number, NCT04786262.).
Key findings
- A total of 14 participants (2 in part A and 12 in parts B and C) completed at least 12 months of follow-up and were included in the analyses.
- C-peptide was undetectable at baseline in all 14 participants.
- After zimislecel infusion, all the participants had engraftment and islet function, as evidenced by the detection of C-peptide.
- Neutropenia was the most common serious adverse event, occurring in 3 participants.
- Two deaths occurred - one caused by cryptococcal meningitis and one by severe dementia with agitation owing to the progression of preexisting neurocognitive impairment.
Why it matters
This small, short-term study tested a stem cell-derived islet infusion in 14 people with type 1 diabetes and found that most no longer needed insulin, but it was not a study of Alzheimer's disease treatment or prevention. One participant died of severe dementia with agitation related to preexisting neurocognitive impairment, which was reported as a death in the study rather than a dementia outcome being tested.
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Study details
- Participants14
- Evidence strengthLow
How this study scored 62/100
Published in a top-tier journal. Scored zero for study design, effect reported and randomised / blinded because the record does not state it.
| Component | Points | Why |
|---|---|---|
| Journal tier | 10 / 10 | Tier 1 — top-tier general journal (New England Journal of Medicine) |
| Journal's citation record | 4 / 4 | New England Journal of Medicine: journal h-index 1,505 (OpenAlex, 2026-10-29) |
| Study design | 0 / 12 | No study design recorded — scores zero, not guessed (not stated in the record — scored 0) |
| Strength of evidence | 1 / 5 | Rated “low” in our summary |
| Peer-review validation | 10 / 10 | Peer-reviewed. Published as an article in a named peer-reviewed journal. |
| How recent | 4 / 5 | 2025 — within 3 year(s) of the newest study here |
| Sample size | 1 / 4 | 14 participants |
| Effect reported | 0 / 3 | No effect size reported — scores zero (not stated in the record — scored 0) |
| Studied in people | 3 / 3 | Studied in people (participant count stated) |
| Randomised / blinded | 0 / 2 | No randomisation or blinding stated — scores zero (not stated in the record — scored 0) |
| Times cited | 5 / 7 | Cited 165 times (OpenAlex, 2026-10-10) |
| Citations per year | 5 / 5 | 55.0 citations a year over 3 year(s) since 2025 |
| Impact for its field and year | 6 / 6 | Cited 145.8× the average for papers of its field and year (OpenAlex FWCI) |
| Cited by studies here | 0 / 6 | No other study in this library cites it |
| Still being cited | 3 / 4 | Cited 165 times in 2024–2026 (2026 still in progress; OpenAlex) |
| Senior author's track record | 3 / 4 | Last-listed author Michael Roehrhoff Rickels: h-index 62 (OpenAlex, whole career) |
| Institution's track record | 2 / 3 | University of Toronto: institution h-index 1,923 (OpenAlex) |
| Multi-centre collaboration | 3 / 3 | OpenAlex lists 15 institution(s) in 4 countries |
| Indexed in PubMed | 2 / 2 | Indexed in PubMed (PMID 40544428) |
| Free to read | 0 / 2 | Behind a paywall — scores zero |
| Total | 62 / 100 | — |
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Read the original
May need a subscription OpenAlex found no free copy of the full text (checked 6 Feb 1020), so the publisher may charge for it. The PubMed abstract is free.
Cite this study APA, Vancouver, BibTeX · download .bib or .ris
Built from the record alzhub holds for this paper, not looked up. Volume, issue and page numbers are not held in the record. The record's author list ends in “et al.”, so not every author is named. Check the citation against the original before you use it.
APA 7th
Reichman, T., Markmann, J. F., Odorico, J. S., Witkowski, P., Fung, J. J., Wijkstrom, M. N., et al. (2025). Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes. New England Journal of Medicine. https://doi.org/10.1056/nejmoa2506549
Vancouver
Reichman T, Markmann JF, Odorico JS, Witkowski P, Fung JJ, Wijkstrom MN, et al. Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes. New England Journal of Medicine. 2025. doi:10.1056/nejmoa2506549. PMID: 40544428.
BibTeX
@article{reichman2025-40544428,
author = {Reichman, Trevor and Markmann, James F. and Odorico, Jon S. and Witkowski, Piotr and Fung, John J. and Wijkstrom, Martin N. and Kandeel, Fouad F and de Koning, Eelco J.P. and others},
title = {{Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes}},
journal = {New England Journal of Medicine},
year = {2025},
doi = {10.1056/nejmoa2506549},
pmid = {40544428},
url = {https://doi.org/10.1056/nejmoa2506549}
}
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