Disease Briefing

Chronic myeloid leukemia: research and therapeutic landscape

A live synthesis of what is being targeted, what is being trialled, which datasets exist, and where the actionable gaps sit — assembled from public APIs, with every figure traceable to its source.

Generated 2026-09-30Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 174 studies · 9,227 GEO samples
Page Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in chronic myeloid leukemia — ABL1, BCR, LYN, SRC, ASXL1, RUNX1, IKZF1, TP53, BCL2, STAT5A, JAK2, ABCB1 — triaged for what can actually be aimed at them, alongside the trials, public datasets, literature and funding around the disease. Every figure below carries its source and retrieval date in the section it comes from.

8
drugs carry an FDA label naming chronic myeloid leukemia: Asciminib, Bosutinib, Dasatinib, Hydroxyurea, Imatinib, Nilotinib and 2 more. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
4
of the 12 genes above carry a drug that is approved in chronic myeloid leukemia itself — ABL1, BCR, LYN, SRC. Across all of them 114 drug entries reach these genes, 91 distinct once salt forms are merged
0
registered BCR-ABL1 cell-therapy trials in chronic myeloid leukemia, 0 active. Counted from ClinicalTrials.gov across 16 synonyms, deduplicated by NCT id, interventional studies only — the query is published in the JSON beside this page so the number can be re-derived
4
targets carry an Open Targets tractability signal and no trial in chronic myeloid leukemia that the searches found (no trial naming a drug Open Targets lists for the gene, none naming it as a cell-therapy target): ASXL1, RUNX1, IKZF1, STAT5A
3,342
human GEO series match the disease; 174 survive on-topic filtering, and only 5 are patient cohorts of 100+ samples
46
Europe PMC full-text papers name GSE4170 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$11.1M
NIH obligations in FY2025, down 62% since 2013 — while distinct core projects went 39 to 18. Larger awards rather than more distinct core projects; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-30 · weekly

12 genes recurrently implicated in chronic myeloid leukemia, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 8 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Chronic myeloid leukemia does have labelled therapy — 8 drugs, listed in the next section.

Two different questions

The table below asks what can be aimed at 12 recurrently implicated genes. That is narrower than what is approved for the disease, and narrower again than what chronic myeloid leukemia is actually treated with, which includes agents approved under broader indications. All three are on this page and they are kept apart deliberately.

GeneDrugsApproval, and whether it reached this diseaseOpen Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it worksCell therapy here
ABL1 across cancers → 20 5 approvedAsciminib, Bosutinib, Nilotinib, Ponatinib, UmbralisibApproved in chronic myeloid leukemia: Asciminib, Bosutinib, Nilotinib, Ponatinib.88 active of 479 chronic myeloid leukemia trials antibody, protein degrader, small molecule —
BCR 9 4 approvedAsciminib, Bosutinib, Nilotinib, PonatinibApproved in chronic myeloid leukemia: Asciminib, Bosutinib, Nilotinib, Ponatinib.86 active of 464 chronic myeloid leukemia trials antibody, protein degrader, small molecule —
LYN 9 1 approvedBosutinibApproved in chronic myeloid leukemia: Bosutinib.26 active of 131 chronic myeloid leukemia trials antibody, protein degrader, small molecule —
SRC 11 1 approvedBosutinibApproved in chronic myeloid leukemia: Bosutinib.26 active of 130 chronic myeloid leukemia trials antibody, protein degrader, small molecule —
ASXL1 across cancers → 0 No drug— protein degrader —
RUNX1 across cancers → 0 No drug— protein degrader, small molecule —
IKZF1 across cancers → 0 No drug— protein degrader, small molecule —
TP53 across cancers → 8 Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, Teprasiran1 trials, none active other clinical modality, protein degrader, small molecule —
BCL2 across cancers → 5 2 approvedNavitoclax, Oblimersen6 active of 15 chronic myeloid leukemia trials antibody, other clinical modality, protein degrader, small molecule —
STAT5A 0 No drug— protein degrader, small molecule —
JAK2 across cancers → 24 10 approvedDelgocitinib, Deuruxolitinib, Fedratinib, Filgotinib, Lestaurtinib, Momelotinib, Pacritinib, Ruxolitinib, Tofacitinib, UpadacitinibApproved in atopic eczema, dermatitis, alopecia areata and 8 other indications. No chronic myeloid leukemia indication appears on these drugs’ labels.5 active of 15 chronic myeloid leukemia trials antibody, protein degrader, small molecule —
ABCB1 5 Phase 3Biricodar, Encequidar, Tariquidar, Valspodar, Zosuquidar1 trials, none active antibody, other clinical modality, protein degrader, small molecule —

Dataset evidence counts studies in the 174-study ranked set whose title or abstract names the gene; the bar is scaled to BCR. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-30. Clinical stage is the highest reached for any indication — see the per-drug indications noted in each row. Target-prioritisation reference only; not clinical or prescribing guidance.

What is actually approved here

openFDA drug labels · weekly

8 drugs carry an FDA label naming chronic myeloid leukemia: Asciminib, Bosutinib, Dasatinib, Hydroxyurea, Imatinib, Nilotinib, Ponatinib, Thioguanine. Separately, 13 of the drugs returned for the genes in the table above are approved only for other diseases and reach chronic myeloid leukemia through trials, not through their labels.

8Labelled for chronic myeloid leukemiaFDA INDICATIONS AND USAGE names the disease
13Approved, but for another diseasereturned for the genes in the table above
1Backbone agents listing itbroad cytotoxics whose labels name many tumours

Every label that names chronic myeloid leukemia

DrugRoleWhat the label says
AsciminibSCEMBLIXLabelled herePh+ CML in CP with the T315I mutation.
BosutinibBOSULIF, BosutinibLabelled here( 1 ) adult patients with accelerated, or blast phase Ph+ CML with resistance or intolerance to prior therapy.
DasatinibDASATINIB, Dasatinib, PHYRAGOLabelled here(1 , 14) pediatric patients 1 year of age and older with Ph+ CML in chronic phase.
HydroxyureaHYDREA, HYDROXYUREA, HydroxyureaLabelled hereHYDREA is an antimetabolite indicated for the treatment of: Resistant chronic myeloid leukemia.
ImatinibGleevec, IMATINIB MESYLATE, IMKELDILabelled herePh+ CML in Blast Crisis (BC), Accelerated Phase (AP) or Chronic Phase (CP) After Interferon-alpha (IFN) Therapy Patients with Philadelphia chromosome positive chronic myeloid leukemia in blast crisis, accelerated phase, or in chronic phase after failure of interferon-alpha therapy.
NilotinibCAVHANZA, DANZITEN, NilotinibLabelled hereAdult Patients with Newly Diagnosed Ph+ CML-CP CAVHANZA is indicated for the treatment of adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase.
PonatinibIclusigLabelled hereChronic Myeloid Leukemia (CML) Chronic phase (CP) CML with resistance or intolerance to at least two prior kinase inhibitors.
ThioguanineTABLOIDLabelled hereAlthough thioguanine is one of several agents with activity in the treatment of the chronic phase of chronic myelogenous leukemia, more objective responses are observed with MYLERAN ® (busulfan), and therefore busulfan is usually regarded as the preferred drug.
BusulfanBUSULFAN, BUSULFEX, BusulfanBackboneBUSULFEX is an alkylating drug indicated for: Use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia (CML) ( 1 )

83 labels carry a positive indication naming the disease under indications_and_usage:"chronic myeloid leukemia" OR indications_and_usage:"chronic myelogenous leukemia" OR indications_and_usage:"Ph+ CML" OR indications_and_usage:"Philadelphia chromosome-positive chronic"; they collapse to 9 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-18. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy

ClinicalTrials.gov · retrieved 2026-09-18 · weekly

No cell-therapy trial registered in this disease names one of the targets this briefing searched. That is a bounded finding, not proof of absence: the targets searched and the synonyms used are listed in the machine-readable facts, and a trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the chronic myeloid leukemia literature, not a count, because the field itself grew: 2015–2018 (n=2,450) against 2021–2025 (n=2,238). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Leukemia, Myeloid0.2%5.27%25.84×118 papers
Niacinamide0.33%3.89%11.91×87 papers
Chronic Disease0.49%5.0%10.22×112 papers
Pyrazoles0.73%4.38%5.96×98 papers
Proto-Oncogene Proteins c-abl0.49%1.43%2.92×32 papers
Genomics0.2%0.58%2.85×13 papers
Molecular Docking Simulation0.45%0.94%2.09×21 papers
Cross-Sectional Studies0.86%1.79%2.09×40 papers
Myeloproliferative Disorders0.9%1.56%1.74×35 papers
Tumor Microenvironment0.82%1.39%1.7×31 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Disease-Free Survival5.39%0.63%0.12×14 papers
Survival Analysis3.55%0.54%0.15×12 papers
Tumor Cells, Cultured3.31%0.58%0.18×13 papers
Protein-Tyrosine Kinases8.53%1.88%0.22×42 papers
Gene Expression Regulation, Neoplastic3.84%0.98%0.26×22 papers
Neoplasm Proteins2.98%0.8%0.27×18 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Protein Kinase Inhibitors37.59%49.78%1.32×1114 papers
Fusion Proteins, bcr-abl27.76%29.67%1.07×664 papers
Imatinib Mesylate31.31%27.66%0.88×619 papers
Antineoplastic Agents34.29%26.23%0.77×587 papers
Drug Resistance, Neoplasm16.86%19.48%1.16×436 papers
Treatment Outcome19.1%14.08%0.74×315 papers
Apoptosis10.24%10.05%0.98×225 papers
Dasatinib11.39%9.47%0.83×212 papers
K562 Cells11.1%9.07%0.82×203 papers
Tyrosine Kinase Inhibitors0.0%8.8%new197 papers

Publication mix

Type2015–182021–25
Clinical Trial0.0%0.2%
Randomized Controlled Trial1.7%1.5%
Review13.4%11.4%
Meta-Analysis0.7%0.5%
Case Reports0.0%2.5%

Query: Leukemia, Myelogenous, Chronic, BCR-ABL Positive[MeSH Major Topic] NOT ("Leukemia, Myelomonocytic, Chronic"[MeSH] OR "Precursor B-Cell Lymphoblastic Leukemia-Lymphoma"[MeSH] OR "Primary Myelofibrosis"[MeSH] OR "Polycythemia Vera"[MeSH] OR "Thrombocythemia, Essential"[MeSH]). Terms need at least 12 papers in the recent window and, in the rising table, at least 5 in the earlier one — a term going from 1 paper to 12 is a large fold change and no evidence of anything. Ubiquitous descriptors (Humans, Animals, age bands) and the disease’s own term are dropped. Source: PubMed MeSH, retrieved 2026-09-12.

Disease burden

What the public sources count, and how closely each category matches this disease. The same figures for every disease on the site are on one table.

MeasureValueMatchWhat it counts
New cases each year9,650 cases/yeardirect2026
Deaths each year1,170 deaths/yeardirect2026
Incidence rate2.1 cases per 100,000 people per yeardirect2019-2023
Death rate0.3 deaths per 100,000 people per yeardirect2020-2024
People living with it76,692 people living with the diseasedirect2023
Median age at diagnosis67.0 yearsdirect2019-2023
median age at death77 yearsdirect2020-2024
Five-year relative survival71.1%direct2016-2022

Years of life lost (proxy)

3.3 years per case, 31,793 a year, from survival and age at diagnosis rather than from a death count. derived proxy Not the WHO/GBD measure, which uses age at death and a life table; comparable across these briefings, not with published YLL figures. What this costs to fund is in Funding.

3 assumptions behind this estimate
  • Five-year relative survival stands in for cure; a death after five years is not counted.
  • The median age at diagnosis stands in for the whole age distribution.
  • Life expectancy at birth (78.4 years) is the reference, rather than remaining expectancy at the age of death from a life table.
  • Each assumption moves the result by more than the difference between the two diseases currently on this site, so this figure separates a disease from one ten times its size, not from a close neighbour.

Funding

NIH RePORTER · quarterly

NIH obligations naming chronic myeloid leukemia, after removing the 2,768 records across all years that matched the search but are about something else. That filter is not cosmetic: without it this section reports another field’s funding as this disease’s.

$11.1MNIH obligations, FY2025from $29.0M in FY2013 · -62%
18distinct projects funded39 in FY2013
$29.0Mpeak year was FY2013obligations, all institutes
79%of FY2025 awards from NCI15 of 19

NIH obligations by fiscal year

$7M$15M$22M$29M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$29.0M4639213
FY2014$28.8M4639224
FY2015$11.3M3636231
FY2016$11.1M3232248
FY2017$10.9M2929255
FY2018$10.2M2121182
FY2019$10.6M2727196
FY2020$7.1M2322204
FY2021$7.5M2019213
FY2022$5.9M1717195
FY2023$5.7M1818214
FY2024$7.1M1818203
FY2025$11.1M1918190

Where FY2025 money went

InstitutionObligationsAwards
Seattle Children'S Hospital$3.5M1
Northwestern University$1.1M2
Beckman Research Institute/City Of Hope$1.1M2
University Of Alabama At Birmingham$0.9M2
University Of California-Irvine$0.9M2
H. Lee Moffitt Cancer Ctr & Res Inst$0.7M1
University Of Pittsburgh At Pittsburgh$0.6M1
University Of Arizona$0.5M1
Case Western Reserve University$0.4M0
Baylor College Of Medicine$0.4M1

Text search chronic myeloid leukemia over project title, terms and abstract, per fiscal year. Awards are individual funding actions; distinct projects collapse them by core project number, so a multi-year grant counts once. The most recent year may be incomplete. Source: NIH RePORTER, retrieved 2026-09-19.

What that buys

Against 31,793 years of life lost a year, FY2025 obligations are $349 per life-year — $1,150 per case. The estimate and its assumptions are in Disease burden.

Who funds it, FY2025

The total says how much. These say who from, through what kind of award, and to whom — which a dollar figure cannot.

NIH institutes

NCI15 · 79%
NHLBI4 · 21%

Projects by administering institute. The rows above are the top 2 and account for 19 of 19.

Award mechanisms

R0115 · 79%
P011 · 5%
P301 · 5%
K991 · 5%
F301 · 5%

R01 is an investigator-initiated grant; ZIA is NIH intramural, meaning the work happens inside NIH rather than being funded outside it. The rows above are the top 5 and account for 19 of 19.

Where it lands

Seattle Children'S Hospital$3.5M · 31.4%
Northwestern University$1.1M · 10.1%
Beckman Research Institute/City Of Hope$1.1M · 10.0%
University Of Alabama At Birmingham$0.9M · 8.0%
University Of California-Irvine$0.9M · 7.8%
H. Lee Moffitt Cancer Ctr & Res Inst$0.7M · 6.0%

Share of $11.1M in FY2025. The top three hold 52%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

3,342 human GEO series match chronic myeloid leukemia. Keyword relevance cannot tell a 2,333-sample patient cohort from a six-well cell-line experiment, so this ranking scores four things GEO does not expose: how often the accession is named in full-text papers, field-normalised citation impact, clinical annotation, and cohort type.

Composition of the 174-study ranked set

patient 79cell line 39mixed 32unspecified 22xenograft 2
79 patient39 cell line32 mixed22 unspecified2 xenograft127 carry clinical annotation38 carry survival5 patient cohorts ≥100 GEO samples9,227 GEO samples totalin 8 single-cell series the GEO sample count is the submitter’s unit (cells, libraries or pools), not patients

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE4170Gene expression changes associated with progression and response in chronic myeloid leukemia2006 · array119468.8
patient cohortAPT 0.95molecularIMATINIB464 cites
GSE76312Single-cell transcriptomics uncovers distinct molecular signatures of stem cells in chronic myeloid leukemia2017 · single-cell2,289289.0
APT 0.75334 cites
GSE16432Musashi-2 regulates normal hematopoiesis and promotes aggressive myeloid leukemia2010 · array436106.2
mixedAPT 0.75survivalmolecularABL1BCRBCR-ABL1334 cites
GSE130404Gene expression signature that predicts early molecular response failure in chronic phase CML patients on frontline imatinib2019 · array96111.4
patient cohortAPT 0.75survivalstagemolecularABL1BCRBCR-ABL136 cites
GSE14671EXPRESSION SIGNATURE TO PREDICT MAJOR CYTOGENETIC RESPONSE IN CHRONIC PHASE CML PATIENTS TREATED WITH IMATINIB2010 · array59225.4
patient cohortAPT 0.75survivalmolecularIMATINIB196 cites
GSE47927Comparing gene expression in stem/progenitor cells from patients with CML in chronic, accelerated and blastic phase with normal volunteers2013 · array67345.4
patient cohortAPT 0.75196 cites
GSE5550Molecular signature of CD34+ hematopoietic stem and progenitor cells in chronic phase CML2006 · array17255.4
mixedAPT 0.75molecularBCRBCR-ABL196 cites
GSE62121Charaterization of genetic alterations and gene expression signatures found in BCR-ABL inhibitor-resistant KCL-22 subpopulations and single clones2014 · sequencing818.8
mixedAPT 0.75survivalmolecularABL1BCRBCR-ABL382 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE236233Single cell multi-omics analysis of chronic myeloid leukemia links cellular heterogeneity to therapy response2024 · sequencing1734.8
patient cohortAPT 0.75molecularABL1BCRBCR-ABL37 cites
GSE276122Combined targeting of GPX4 and BCR-ABL tyrosine kinase selectively compromises BCR-ABL+ leukemia stem cells2024 · sequencing414.3
mixedAPT 0.5survivalmolecularBCRBCR-ABL34 cites
GSE226360Targeting FLT3-TAZ Signaling to Suppress Drug Resistance in Blast Crisis Chronic Myeloid Leukemia2023 · sequencing1211.4
patient cohortAPT 0.25survivalmolecularABL1BCRBCR-ABL116 cites
GSE216837Pyruvate Anaplerosis is a Targetable Vulnerability in Persistent Leukaemic Stem Cells2023 · sequencing811.9
patient cohortAPT 0.25survivalmolecularBCRBCR-ABLIMATINIB21 cites
GSE218451BCR-ABL triggers a glucose-dependent survival program during leukemogenesis through the suppression of TXNIP2023 · sequencing911.8
patient cohortAPT 0.05survivalmolecularBCRBCR-ABLIMATINIB18 cites
GSE279135Bone marrow CD34+/lin- cells of patients with Chronic Phase- Chronic Myeloid Leukemia (CP-CML) after 12 months of nilotinib treatment exhibit a different expression signature compared to the diagnosis and the same cells from healthy subjects2025 · array1681—
patient cohortAPT 0.05survivalmolecularABL1BCRBCR-ABL1

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-19. SubSeries are collapsed to one row per study by linked PMID. Of 3,331 series retrieved, 110 were dropped by the profile’s exclusion rules and 3,033 named the disease only in passing. The ranked set is a relevance-ranked sample, not a census, so the composition figures describe the sample only.

Where the gaps are

Derived from the sections above · recomputed on every refresh

Each card below is a disagreement between two of the tables on this page — a drug that exists but was never tried here, a target that looks tested and was not, a gene with no drug whose product has a busy clinical programme. They are computed from the same facts as the tables, so they cannot contradict them.

ASXL1

Open Targets lists no drug against it and no trial in chronic myeloid leukemia names it. A bounded negative: the sources and search terms are in the JSON beside this page.

RUNX1

Open Targets lists no drug against it and no trial in chronic myeloid leukemia names it. A bounded negative: the sources and search terms are in the JSON beside this page.

IKZF1

Open Targets lists no drug against it and no trial in chronic myeloid leukemia names it. A bounded negative: the sources and search terms are in the JSON beside this page.

STAT5A

Open Targets lists no drug against it and no trial in chronic myeloid leukemia names it. A bounded negative: the sources and search terms are in the JSON beside this page.

How a machine reads this page

Static

This page is written to be quoted correctly by a model as much as read by a person. The same facts are available as JSON at the sibling URL below, which is what to cite. The cross-disease layer — burden, genes, drugs and antigens across every briefing — is described at /disease/api.json.

Fully server-rendered

Every figure is in the HTML at first byte. No JavaScript runs, no figure is fetched after load, and nothing on this page requires a renderer to see.

Every number carries a source

Each section names the API it came from and the date it was retrieved: Open Targets (2026-09-30), ClinicalTrials.gov (2026-09-18), openFDA (2026-09-18), NCBI GEO (2026-09-19), PubMed (2026-09-12), NIH RePORTER (2026-09-19).

Negatives stated explicitly

Where the searches find nothing for chronic myeloid leukemia — no drug, no trial, no approval — the page says so in those words rather than omitting the row, and names the source searched. An absent row is unquotable; a bounded negative is the finding.

Denominators, not just percentages

Every share is printed with the count and the total it came from, so a figure can be checked rather than taken.

The filters are published

3 exclusion patterns are applied to free text before anything is ranked, because K562 is used as a model system in NK-cell cytotoxicity assays, erythroid and megakaryocytic differentiation, generic haematopoietic cell biology. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Chronic myeloid leukemia",
  "mesh": "Leukemia, Myelogenous, Chronic, BCR-ABL Positive",
  "facts": "https://usebiotransfer.org/disease/chronic-myeloid-leukemia.json",
  "methods": "https://usebiotransfer.org/methods/",
  "all_diseases": "https://usebiotransfer.org/disease/api.json",
  "note": "Every figure on the page is in that JSON with its source and retrieval date. Quote from it rather than from the HTML."
}