Imatinib (tyrosine kinase inhibitor)
Novartis (Gleevec/Glivec; repurposed)
What it is
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.
Editorial review: .
Most recent recorded citation date: 2022-07-15. Only explicit date metadata is included; an undated citation may be newer. This does not mean every claim was reviewed on that date.
Trial status, labels and access can change between reviews. How we review the evidence · How to read the evidence
Evidence behind this assessment
Key evidence notes. Study results, product eligibility and access answer different questions.
- Who was studied?
- Study populations and analysis groups vary. Product age limits alone do not describe who was studied.See the linked studies and their populations →
- Benefit or performance
- Delay of onset: Preserved C-peptide at 12 months in recent-onset adults, but has not shown onset prevention.[1]
- Important harms and treatment burden
- Safety: Adverse events were common enough to make chronic use hard to justify in otherwise healthy-feeling people without stronger benefit.[1]
- Approval and country access
- Country-specific approval and access are not summarized in this record.Approval, trial recruitment, local supply and funding are separate. Check the cited label or access source.
- Follow-up and remaining uncertainty
- Durability: The 12-month C-peptide benefit was not sustained at 24 months after the 26-week treatment course ended.[1]
Research status alone does not establish approval, clinical benefit or local availability.
Editorial score: calculation and evidence
A weighted editorial judgment on a 0–100 scale, not a probability of success or a measured treatment effect. Higher criterion scores mean more favorable assessments.
Default calculation: 42 × 30 + 25 × 20 + 35 × 20 + 25 × 15 + 30 × 15 = 3285; divide by total weight 100. Unrounded weighted result: 32.85.
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]
Oncology-market availability notwithstanding, the cited FDA label covers oncology indications with no T1D indication, and the trial safety/tolerability profile limits 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.
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 study · 2021-06-29 — PMID 34214479. NCT01781975. Adults 18-45 within 100 days of diagnosis; met 12-month C-peptide endpoint; effect not sustained at 24 months.
Gitelman SE, et al. Imatinib therapy for patients with recent-onset type 1 diabetes. Lancet Diabetes & Endocrinology (2021).
- [2]Imatinib Treatment in Recent Onset Type 1 Diabetes Mellitus · Trial registry · 2020-02-11 — Completed phase 2; actual enrollment 67.
- [3]GLEEVEC (imatinib mesylate) prescribing information · Regulatory decision · 2022-07-15 — FDA label for oncology indications; no T1D indication.