Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes
Authors' conclusion
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.
Study context
- Population
- Persons with T1D; all received glucocorticoid-free immunosuppressive therapy≥12 months follow-up
- Participants
- 14 persons
- Comparison
- Zimislecel half dose and full dose
- Part An = 2
- Parts B and Cn = 12
- Outcome
- Part C: freedom from severe hypoglycemic events (days 90–365) with HbA1c <7% or decrease ≥1 percentage point from baseline at ≥1 time point between days 180 and 365; part A: safety
- Follow-up
- Days 90 through 365
Methodological detailsinterim analyses, not prespecified
- Interim analyses
- Not prespecified
Main finding
As reported in the abstract
Free of severe hypoglycemic events and HbA1c <7%
Parts B and C
12
events
- All 12 participants in parts B and C met the primary end point
- 12 of 12
Secondary outcomes
Insulin independence (no exogenous insulin) · day 365
- Ten of the 12 participants in parts B and C had insulin independence and were not using exogenous insulin
- 10 of 12 (83%)
- Interim analysis, not prespecified
Time in target glucose range (70 to 180 mg/dL)
- All 12 participants in parts B and C spent >70% of time in target range
Engraftment and islet function (C-peptide detection during mixed-meal tolerance test)
- C-peptide undetectable at baseline in all participants
- 14
- All participants had engraftment and islet function, as evidenced by C-peptide detection
Safety
- Neutropenia, most common serious adverse event
- 3 participants
- Deaths (1 cryptococcal meningitis; 1 severe dementia with agitation from progression of preexisting neurocognitive impairment)
- 2
Original publication
Stem Cell–Derived, Fully Differentiated Islets for Type 1 Diabetes. N Engl J Med. Published 20 June 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →