Imatinib (tyrosine kinase inhibitor)
Novartis (Gleevec/Glivec; repurposed)
A repurposed oral cancer drug tested in adults within 100 days of T1D diagnosis. A phase-2 trial met its 12-month C-peptide endpoint, but the effect was not sustained at 24 months after a 26-week course and adverse events were common. Not approved for T1D.
The scorecard
Preserved C-peptide at 12 months in recent-onset adults, but has not shown onset prevention.[1]
The 12-month C-peptide benefit was not sustained at 24 months after the 26-week treatment course ended.[1]
Adverse events were common enough to make chronic use hard to justify in otherwise healthy-feeling people without stronger benefit.[1]
Generic oncology drug availability helps, but there is no T1D indication and safety/tolerability limit practical prevention use.[3]
Editor’s take
Imatinib is a useful cautionary record: it proves some repurposed oral drugs can move C-peptide, but short-course benefit that fades by 24 months is not enough. It belongs in the map so readers can see why "positive phase 2" is not the same as a viable prevention therapy.
The full picture
Evidence
Imatinib is a tyrosine kinase inhibitor best known as an oncology drug. In T1D, it was tested because preclinical work suggested it could affect immune and metabolic pathways relevant to beta-cell survival.
The phase-2 trial enrolled adults aged 18-45 within 100 days of diagnosis. It met the 12-month C-peptide endpoint, but the benefit did not hold through 24 months after stopping treatment.1 That makes it a real signal, but a weak durability story.
References
-
Gitelman SE, et al. Imatinib therapy for patients with recent-onset type 1 diabetes. Lancet Diabetes & Endocrinology (2021). https://doi.org/10.1016/S2213-8587%2821%2900139-X ↩
Coming soon
ETA · No active pivotal T1D program found in this pass
Sources
- [1]Imatinib therapy for patients with recent-onset type 1 diabetes: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial · peer-reviewed · 2021-07-05 — PMID 34214479. NCT01781975. Adults 18-45 within 100 days of diagnosis; met 12-month C-peptide endpoint; effect not sustained at 24 months.
- [2]Imatinib Treatment in Recent Onset Type 1 Diabetes Mellitus · registry · 2020-02-11 — Completed phase 2; actual enrollment 67.
- [3]GLEEVEC (imatinib mesylate) prescribing information · regulatory · 2022-07-15 — FDA label for oncology indications; no T1D indication.