Target landscape
Open Targets · retrieved 2026-09-30 · weekly12 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.
| Gene | Drugs | Approval, and whether it reached this disease | Open Targets tractabilitycomputed from protein features and literature — a signal that a modality is worth trying, not evidence that it works | Cell 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 · weekly8 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.
Every label that names chronic myeloid leukemia
| Drug | Role | What the label says |
|---|---|---|
| AsciminibSCEMBLIX | Labelled here | Ph+ CML in CP with the T315I mutation. |
| BosutinibBOSULIF, Bosutinib | Labelled here | ( 1 ) adult patients with accelerated, or blast phase Ph+ CML with resistance or intolerance to prior therapy. |
| DasatinibDASATINIB, Dasatinib, PHYRAGO | Labelled here | (1 , 14) pediatric patients 1 year of age and older with Ph+ CML in chronic phase. |
| HydroxyureaHYDREA, HYDROXYUREA, Hydroxyurea | Labelled here | HYDREA is an antimetabolite indicated for the treatment of: Resistant chronic myeloid leukemia. |
| ImatinibGleevec, IMATINIB MESYLATE, IMKELDI | Labelled here | Ph+ 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, Nilotinib | Labelled here | Adult 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. |
| PonatinibIclusig | Labelled here | Chronic Myeloid Leukemia (CML) Chronic phase (CP) CML with resistance or intolerance to at least two prior kinase inhibitors. |
| ThioguanineTABLOID | Labelled here | Although 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, Busulfan | Backbone | BUSULFEX 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 · weeklyNo 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 · monthlyEvery 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 term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Leukemia, Myeloid | 0.2% | 5.27% | 25.84× | 118 papers |
| Niacinamide | 0.33% | 3.89% | 11.91× | 87 papers |
| Chronic Disease | 0.49% | 5.0% | 10.22× | 112 papers |
| Pyrazoles | 0.73% | 4.38% | 5.96× | 98 papers |
| Proto-Oncogene Proteins c-abl | 0.49% | 1.43% | 2.92× | 32 papers |
| Genomics | 0.2% | 0.58% | 2.85× | 13 papers |
| Molecular Docking Simulation | 0.45% | 0.94% | 2.09× | 21 papers |
| Cross-Sectional Studies | 0.86% | 1.79% | 2.09× | 40 papers |
| Myeloproliferative Disorders | 0.9% | 1.56% | 1.74× | 35 papers |
| Tumor Microenvironment | 0.82% | 1.39% | 1.7× | 31 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Disease-Free Survival | 5.39% | 0.63% | 0.12× | 14 papers |
| Survival Analysis | 3.55% | 0.54% | 0.15× | 12 papers |
| Tumor Cells, Cultured | 3.31% | 0.58% | 0.18× | 13 papers |
| Protein-Tyrosine Kinases | 8.53% | 1.88% | 0.22× | 42 papers |
| Gene Expression Regulation, Neoplastic | 3.84% | 0.98% | 0.26× | 22 papers |
| Neoplasm Proteins | 2.98% | 0.8% | 0.27× | 18 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Protein Kinase Inhibitors | 37.59% | 49.78% | 1.32× | 1114 papers |
| Fusion Proteins, bcr-abl | 27.76% | 29.67% | 1.07× | 664 papers |
| Imatinib Mesylate | 31.31% | 27.66% | 0.88× | 619 papers |
| Antineoplastic Agents | 34.29% | 26.23% | 0.77× | 587 papers |
| Drug Resistance, Neoplasm | 16.86% | 19.48% | 1.16× | 436 papers |
| Treatment Outcome | 19.1% | 14.08% | 0.74× | 315 papers |
| Apoptosis | 10.24% | 10.05% | 0.98× | 225 papers |
| Dasatinib | 11.39% | 9.47% | 0.83× | 212 papers |
| K562 Cells | 11.1% | 9.07% | 0.82× | 203 papers |
| Tyrosine Kinase Inhibitors | 0.0% | 8.8% | new | 197 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Clinical Trial | 0.0% | 0.2% |
| Randomized Controlled Trial | 1.7% | 1.5% |
| Review | 13.4% | 11.4% |
| Meta-Analysis | 0.7% | 0.5% |
| Case Reports | 0.0% | 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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | 9,650 cases/year | direct | 2026 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| Deaths each year | 1,170 deaths/year | direct | 2026 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| Incidence rate | 2.1 cases per 100,000 people per year | direct | 2019-2023 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| Death rate | 0.3 deaths per 100,000 people per year | direct | 2020-2024 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| People living with it | 76,692 people living with the disease | direct | 2023 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| Median age at diagnosis | 67.0 years | direct | 2019-2023 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| median age at death | 77 years | direct | 2020-2024 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
| Five-year relative survival | 71.1% | direct | 2016-2022 SEER Cancer Stat Facts, Chronic Myeloid Leukemia, retrieved 2026-09-12 |
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 · quarterlyNIH 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.
NIH obligations by fiscal year
| Year | Obligations | Awards | Distinct projects | Dropped as off-topic |
|---|---|---|---|---|
| FY2013 | $29.0M | 46 | 39 | 213 |
| FY2014 | $28.8M | 46 | 39 | 224 |
| FY2015 | $11.3M | 36 | 36 | 231 |
| FY2016 | $11.1M | 32 | 32 | 248 |
| FY2017 | $10.9M | 29 | 29 | 255 |
| FY2018 | $10.2M | 21 | 21 | 182 |
| FY2019 | $10.6M | 27 | 27 | 196 |
| FY2020 | $7.1M | 23 | 22 | 204 |
| FY2021 | $7.5M | 20 | 19 | 213 |
| FY2022 | $5.9M | 17 | 17 | 195 |
| FY2023 | $5.7M | 18 | 18 | 214 |
| FY2024 | $7.1M | 18 | 18 | 203 |
| FY2025 | $11.1M | 19 | 18 | 190 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Seattle Children'S Hospital | $3.5M | 1 |
| Northwestern University | $1.1M | 2 |
| Beckman Research Institute/City Of Hope | $1.1M | 2 |
| University Of Alabama At Birmingham | $0.9M | 2 |
| University Of California-Irvine | $0.9M | 2 |
| H. Lee Moffitt Cancer Ctr & Res Inst | $0.7M | 1 |
| University Of Pittsburgh At Pittsburgh | $0.6M | 1 |
| University Of Arizona | $0.5M | 1 |
| Case Western Reserve University | $0.4M | 0 |
| Baylor College Of Medicine | $0.4M | 1 |
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
Projects by administering institute. The rows above are the top 2 and account for 19 of 19.
Award mechanisms
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
Share of $11.1M in FY2025. The top three hold 52%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly3,342 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
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE4170 | Gene expression changes associated with progression and response in chronic myeloid leukemia2006 · array | 119 | 46 | 8.8 | patient cohortAPT 0.95molecularIMATINIB464 cites |
| GSE76312 | Single-cell transcriptomics uncovers distinct molecular signatures of stem cells in chronic myeloid leukemia2017 · single-cell | 2,289 | 28 | 9.0 | APT 0.75334 cites |
| GSE16432 | Musashi-2 regulates normal hematopoiesis and promotes aggressive myeloid leukemia2010 · array | 436 | 10 | 6.2 | mixedAPT 0.75survivalmolecularABL1BCRBCR-ABL1334 cites |
| GSE130404 | Gene expression signature that predicts early molecular response failure in chronic phase CML patients on frontline imatinib2019 · array | 96 | 11 | 1.4 | patient cohortAPT 0.75survivalstagemolecularABL1BCRBCR-ABL136 cites |
| GSE14671 | EXPRESSION SIGNATURE TO PREDICT MAJOR CYTOGENETIC RESPONSE IN CHRONIC PHASE CML PATIENTS TREATED WITH IMATINIB2010 · array | 59 | 22 | 5.4 | patient cohortAPT 0.75survivalmolecularIMATINIB196 cites |
| GSE47927 | Comparing gene expression in stem/progenitor cells from patients with CML in chronic, accelerated and blastic phase with normal volunteers2013 · array | 67 | 34 | 5.4 | patient cohortAPT 0.75196 cites |
| GSE5550 | Molecular signature of CD34+ hematopoietic stem and progenitor cells in chronic phase CML2006 · array | 17 | 25 | 5.4 | mixedAPT 0.75molecularBCRBCR-ABL196 cites |
| GSE62121 | Charaterization of genetic alterations and gene expression signatures found in BCR-ABL inhibitor-resistant KCL-22 subpopulations and single clones2014 · sequencing | 8 | 1 | 8.8 | mixedAPT 0.75survivalmolecularABL1BCRBCR-ABL382 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE236233 | Single cell multi-omics analysis of chronic myeloid leukemia links cellular heterogeneity to therapy response2024 · sequencing | 17 | 3 | 4.8 | patient cohortAPT 0.75molecularABL1BCRBCR-ABL37 cites |
| GSE276122 | Combined targeting of GPX4 and BCR-ABL tyrosine kinase selectively compromises BCR-ABL+ leukemia stem cells2024 · sequencing | 4 | 1 | 4.3 | mixedAPT 0.5survivalmolecularBCRBCR-ABL34 cites |
| GSE226360 | Targeting FLT3-TAZ Signaling to Suppress Drug Resistance in Blast Crisis Chronic Myeloid Leukemia2023 · sequencing | 12 | 1 | 1.4 | patient cohortAPT 0.25survivalmolecularABL1BCRBCR-ABL116 cites |
| GSE216837 | Pyruvate Anaplerosis is a Targetable Vulnerability in Persistent Leukaemic Stem Cells2023 · sequencing | 8 | 1 | 1.9 | patient cohortAPT 0.25survivalmolecularBCRBCR-ABLIMATINIB21 cites |
| GSE218451 | BCR-ABL triggers a glucose-dependent survival program during leukemogenesis through the suppression of TXNIP2023 · sequencing | 9 | 1 | 1.8 | patient cohortAPT 0.05survivalmolecularBCRBCR-ABLIMATINIB18 cites |
| GSE279135 | Bone 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 · array | 168 | 1 | — | 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 refreshEach 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
StaticThis 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."
}