Drug repurposing for AML

Which approved non-cancer drugs have real published evidence against acute myeloid leukaemia?

Maintained by Igor Moiseev

Public literature and public datasets only. No patient data. No clinical claims. Nothing here is medical advice. If you or someone you care for is affected, talk to the treating physician.

Articles

Why AML, and how this program works
A public, funded literature-and-data program on repurposing approved drugs for acute myeloid leukaemia.
1 Nov 2026
AML drug-repurposing research is spread across many drugs, not concentrated in a few
The pre-registered hypothesis predicted that 5 drugs would account for at least half of the 2015–2025 papers on repurposing approved non-cancer drugs for AML. In Europe PMC they account for about 41–42%, and the long tail holds up under most sensitivity checks.
1 Nov 2026

Research graph

1 experiments · 3 tracks

confirmedpartialrefutedpendingfinding motivated a later hypothesislater experiment controlled an earlier caveatlater experiment superseded an earlier oneEach dot is one experiment, coloured by its finding's verdict; click one to open it.

What runs next

Ranked by value ÷ cost; donations pay for the top one first

01
Did the most-studied candidates reach trials?
Of the 10 most-published repurposing candidates for AML, how many have a registered interventional AML trial on ClinicalTrials.gov?
Pre-registered: Fewer than half of the top-10 candidates have a registered interventional AML trial.
score 2.0value 4 · cost 2
02
Potency versus achievable exposure
For the top candidates, do reported in-vitro IC50 values against AML cell lines fall below the plasma concentrations reached at approved doses?
Pre-registered: For most top candidates the median reported IC50 exceeds the approved-dose Cmax, so in-vitro activity alone overstates clinical promise.
score 1.7value 5 · cost 3

Findings

Latest 1

Refuted
AML drug-repurposing research is spread across many drugs, not concentrated in a few
In the primary strict corpus (Europe PMC 2015–2025, AML plus a repurposing term; 147 papers, 33 matched to a dictionary drug), the top 5 compounds account for 42.4% of drug-assigned papers. The bootstrap 95% CI is 0.39–0.67, so the primary result is below the 50% threshold but not decisive on its own. The larger loose corpus (237 papers, 159 assigned) gives 40.9% (CI 0.35–0.49), which excludes 50%
E:aml-repurposing-11 Nov 2026Provisional

Claims

Each one re-executes on demand

scripts/aml-repurposing-1/analysis.py · script
Europe PMC counts 2015-2025: all AML records; strict AML+repurposing query hits; strict papers kept after filters; loose (drug-in-title) papers kept.
Passed· 5.8s · 15 h ago
scripts/aml-repurposing-1/analysis.py · script
Primary top-5 share of drug-assigned papers (S1): strict 0.4242, loose 0.4088; top-1 share and effective number of compounds.
Passed· 6.5s · 15 h ago
scripts/aml-repurposing-1/analysis.py · script
Sensitivity S1: with ATRA, class-level, preclinical-only scenarios.
Passed· 13.8s · 15 h ago

Runs

E:aml-repurposing-1Is the published preclinical evidence for repurposing approved non-oncology drugs in AML concentrated in a few compounds?done · 1 Nov 2026

Articles

Why AML, and how this program works

AML drug-repurposing research is spread across many drugs, not concentrated in a few