Target landscape
Open Targets · retrieved 2026-09-16 · weekly12 genes recurrently implicated in gastrointestinal stromal tumour, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.
Of 12 recurrently implicated genes, 5 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Gastrointestinal stromal tumour does have labelled therapy — 6 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 gastrointestinal stromal tumour 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 |
|---|---|---|---|---|
| KIT across cancers → | 33 | 15 approvedAvapritinib, Cediranib, Dasatinib, Imatinib, Masitinib, Midostaurin, Pazopanib, Pexidartinib, Quizartinib, Regorafenib, Ripretinib, Sorafenib, Sunitinib, Sunitinib Malate, TivozanibApproved in gastrointestinal stromal tumour: Avapritinib, Imatinib, Pazopanib, Regorafenib, Ripretinib, Sunitinib.68 active of 352 gastrointestinal stromal tumour trials | antibody, protein degrader, small molecule | — |
| PDGFRA across cancers → | 32 | 14 approvedAvapritinib, Becaplermin, Cediranib, Masitinib, Midostaurin, Nintedanib, Nintedanib Esylate, Olaratumab, Pazopanib, Quizartinib, Regorafenib, Ripretinib, Sunitinib, Sunitinib MalateApproved in gastrointestinal stromal tumour: Avapritinib, Pazopanib, Regorafenib, Ripretinib, Sunitinib.43 active of 196 gastrointestinal stromal tumour trials | antibody, other clinical modality, protein degrader, small molecule | — |
| SDHB | 0 | No drug— | antibody, protein degrader, small molecule | — |
| SDHA | 0 | No drug— | antibody, protein degrader, small molecule | — |
| SDHC | 0 | No drug— | antibody, protein degrader, small molecule | — |
| SDHD | 0 | No drug— | antibody, protein degrader, small molecule | — |
| NF1 across cancers → | 0 | No drug— | antibody, protein degrader, small molecule | — |
| BRAF across cancers → | 16 | 6 approvedDabrafenib, Encorafenib, Regorafenib, Sorafenib, Tovorafenib, VemurafenibApproved in gastrointestinal stromal tumour: Regorafenib.12 active of 29 gastrointestinal stromal tumour trials | antibody, protein degrader, small molecule | — |
| ETV1 | 0 | No drug— | protein degrader | — |
| ANO1 | 1 | 1 approvedCrofelemerApproved in Diarrhea. No gastrointestinal stromal tumour indication appears on these drugs’ labels.No gastrointestinal stromal tumour trial of any of these drugs | antibody, protein degrader, small molecule | — |
| CDKN2A across cancers → | 0 | No drug— | — | — |
| TP53 across cancers → | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo gastrointestinal stromal tumour trial of any of these drugs | other clinical modality, protein degrader, small molecule | — |
Dataset evidence counts studies in the 76-study ranked set whose title or abstract names the gene; the bar is scaled to KIT. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-16. 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 · weekly6 drugs carry an FDA label naming gastrointestinal stromal tumour: Avapritinib, Imatinib, Pazopanib, Regorafenib, Ripretinib, Sunitinib. Separately, 18 of the drugs returned for the genes in the table above are approved only for other diseases and reach gastrointestinal stromal tumour through trials, not through their labels.
Every label that names gastrointestinal stromal tumour
| Drug | Role | What the label says |
|---|---|---|
| AvapritinibAyvakit | Labelled here | PDGFRA Exon 18 Mutation-Positive Unresectable or Metastatic Gastrointestinal Stromal Tumor (GIST) AYVAKIT ® is indicated for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations [see Dosage and Administration |
| ImatinibGleevec, IMATINIB MESYLATE, IMKELDI | Labelled here | Kit+ Gastrointestinal Stromal Tumors (GIST) Patients with Kit (CD117) positive unresectable and/or metastatic malignant gastrointestinal stromal tumors. |
| PazopanibPazopanib, VOTRIENT, pazopanib | Labelled here | Limitations of Use : The efficacy of VOTRIENT for the treatment of patients with adipocytic STS or gastrointestinal stromal tumors has not been demonstrated. |
| RegorafenibStivarga | Labelled here | Gastrointestinal Stromal Tumors STIVARGA is indicated for the treatment of adult patients with locally advanced, unresectable or metastatic gastrointestinal stromal tumor (GIST) who have been previously treated with imatinib mesylate and sunitinib malate. |
| RipretinibQINLOCK | Labelled here | QINLOCK is a kinase inhibitor indicated for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. |
| SunitinibSUNITINIB MALATE, SUTENT, Sunitinib Malate | Labelled here | Gastrointestinal Stromal Tumor SUTENT is indicated for the treatment of adult patients with gastrointestinal stromal tumor (GIST) after disease progression on or intolerance to imatinib mesylate. |
0 labels match indications_and_usage:"gastrointestinal stromal tumor" OR indications_and_usage:"gastrointestinal stromal tumors" OR indications_and_usage:"gastrointestinal stromal tumour"; they collapse to 6 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-16. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.
Cell therapy
ClinicalTrials.gov · retrieved 2026-09-16 · weeklyNo cell-therapy trial in this disease names one of the antigens in the profile. That is a finding, not a gap in the query: the antigens searched are listed in the machine-readable facts alongside the synonyms used.
Research momentum
PubMed MeSH · monthlyEvery term below is a share of the gastrointestinal stromal tumour literature, not a count, because the field itself grew: 2015–2018 (n=1,378) against 2021–2025 (n=1,586). 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 |
|---|---|---|---|---|
| Soft Tissue Neoplasms | 0.36% | 1.51% | 4.17× | 24 papers |
| Propensity Score | 0.51% | 1.7% | 3.35× | 27 papers |
| Tumor Microenvironment | 0.36% | 1.2% | 3.3× | 19 papers |
| Progression-Free Survival | 0.58% | 1.64% | 2.82× | 26 papers |
| Nomograms | 0.65% | 1.77% | 2.7× | 28 papers |
| Quality of Life | 0.65% | 1.51% | 2.32× | 24 papers |
| Endoscopic Mucosal Resection | 1.74% | 3.91% | 2.24× | 62 papers |
| Rectum | 1.09% | 2.4% | 2.2× | 38 papers |
| Drug Monitoring | 0.44% | 0.88% | 2.03× | 14 papers |
| Neuroendocrine Tumors | 0.58% | 1.13% | 1.95× | 18 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Neoplasm Staging | 5.88% | 1.01% | 0.17× | 16 papers |
| Time Factors | 5.3% | 1.07% | 0.2× | 17 papers |
| Neoplasm Metastasis | 3.56% | 0.76% | 0.21× | 12 papers |
| Biopsy | 3.77% | 0.82% | 0.22× | 13 papers |
| Disease-Free Survival | 8.85% | 2.02% | 0.23× | 32 papers |
| Tumor Burden | 5.59% | 1.32% | 0.24× | 21 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Gastrointestinal Neoplasms | 36.07% | 27.3% | 0.76× | 433 papers |
| Imatinib Mesylate | 25.76% | 24.4% | 0.95× | 387 papers |
| Stomach Neoplasms | 20.39% | 23.2% | 1.14× | 368 papers |
| Antineoplastic Agents | 24.38% | 22.13% | 0.91× | 351 papers |
| Proto-Oncogene Proteins c-kit | 15.67% | 15.26% | 0.97× | 242 papers |
| Treatment Outcome | 20.46% | 14.19% | 0.69× | 225 papers |
| Mutation | 13.72% | 13.75% | 1.0× | 218 papers |
| Prognosis | 17.85% | 12.8% | 0.72× | 203 papers |
| Protein Kinase Inhibitors | 10.09% | 9.84% | 0.98× | 156 papers |
| Neoplasm Recurrence, Local | 12.26% | 8.64% | 0.7× | 137 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Randomized Controlled Trial | 1.5% | 1.6% |
| Review | 12.3% | 10.0% |
| Meta-Analysis | 1.5% | 1.2% |
| Case Reports | 0.0% | 5.2% |
Query: Gastrointestinal Stromal Tumors[MeSH Major Topic] NOT ("Leiomyoma"[MeSH] OR "Leiomyosarcoma"[MeSH] OR "Mastocytosis"[MeSH] OR "Melanoma"[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 2026-09-16.
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 | 6,000 cases/year | direct | American Cancer Society, Key Statistics for Gastrointestinal Stromal Tumors, retrieved 2026-09-16 |
| Deaths each year | — | not published | Not published on the page; SEER counts GIST deaths under the organ of origin. |
| Five-year relative survival | — | not published | The American Cancer Society publishes five-year relative survival by SEER stage, not one figure; an average would be a derivation the source does not make. |
| Median age at diagnosis | — | not published | Not published as a median: "most often found in people over the age of 50". |
Years of life lost: not computed. Years of life lost needs survival, age at diagnosis and a case count. five_year_relative_survival, median_age_at_diagnosis is not recorded for this disease.
Funding
NIH RePORTER · quarterlyNIH obligations naming gastrointestinal stromal tumour, after removing the 20 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 | $2.3M | 7 | 7 | 3 |
| FY2014 | $1.7M | 8 | 8 | 3 |
| FY2015 | $1.4M | 7 | 7 | 3 |
| FY2016 | $1.7M | 6 | 6 | 3 |
| FY2017 | $3.6M | 7 | 7 | 1 |
| FY2018 | $3.3M | 7 | 7 | 1 |
| FY2019 | $2.8M | 7 | 7 | 1 |
| FY2020 | $8.8M | 11 | 10 | 0 |
| FY2021 | $24.0M | 7 | 7 | 0 |
| FY2022 | $2.4M | 7 | 6 | 2 |
| FY2023 | $7.2M | 6 | 6 | 2 |
| FY2024 | $2.1M | 5 | 5 | 1 |
| FY2025 | $12.1M | 9 | 9 | 0 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Division Of Basic Sciences - Nci | $9.4M | 3 |
| University Of California, San Diego | $1.3M | 2 |
| University Of Pennsylvania | $0.5M | 2 |
| Sloan-Kettering Inst Can Research | $0.5M | 1 |
| Research Inst Of Fox Chase Can Ctr | $0.4M | 1 |
Text search gastrointestinal stromal tumor OR GIST 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 2026-09-16.
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 1 and account for 9 of 9.
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 7 and account for 9 of 9.
Where it lands
Share of $12.1M in FY2025. The top three hold 92%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly165 human GEO series match gastrointestinal stromal tumour. Keyword relevance cannot tell a 415-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 76-study ranked set
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE21123 | Affymetrix SNP array data for soft tissue sarcoma samples2010 · array | 415 | 1 | 12.9 | patient cohortAPT 0.95survivalmolecularKITNF1TP53614 cites |
| GSE107447 | Altered chromosomal topology drives oncogenic programs in SDH-deficient GISTs2019 · chromatin | 100 | 5 | 6.0 | patient cohortAPT 0.75molecularKITPDGFRA210 cites |
| GSE136755 | Driver gene alterations and activated signaling pathways toward malignant progression of gastrointestinal stromal tumors2019 · array | 65 | 15 | 1.1 | patient cohortAPT 0.5stagemolecularKITPDGFRA28 cites |
| GSE56670 | Expression data from SDH-disabled GIST2015 · array | 20 | 5 | 4.4 | patient cohortAPT 0.95molecularSDHASDHBSDHC157 cites |
| GSE34387 | Oncogenic SDH mutation underlies global epigenomic instability in gastrointestinal stromal tumor2013 · methylation | 220 | 3 | 6.9 | APT 0.75molecularKITPDGFRA279 cites |
| GSE8167 | Distinct gene-expression-defined classes of gastrointestinal stromal tumor (GIST).2008 · array | 32 | 16 | 2.1 | APT 0.75molecularKITPDGFRA97 cites |
| GSE15966 | Gene expression signature predictive of rapid response to imatinib mesylate in Gastrointestinal Stromal Tumor (GIST)2009 · array | 54 | 7 | 1.0 | patient cohortAPT 0.5molecularIMATINIB44 cites |
| GSE155800 | Identification of transcriptional difference between Imatinib-sensitive and resistant patients with gastrointerstinal stromal tumor (GIST)2021 · sequencing | 20 | 9 | 0.8 | patient cohortAPT 0.25molecularIMATINIB14 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE254762 | Deciphering the Tumor Immune Microenvironment of Imatinib-Resistance in Advanced Gastrointestinal Stromal Tumors at Single-Cell Resolution2024 · single-cell | 9 | 5 | 3.1 | patient cohortAPT 0.5molecularIMATINIB26 cites |
| GSE151323 | Anatomic and transcriptional determinants of gastrointestinal stromal tumor2022 · sequencing | 9 | 1 | 1.4 | APT 0.5survivalmolecularIMATINIBKIT24 cites |
| GSE172154 | MOZ and Menin-MLL Complexes are Complementary Regulators of Chromatin Association and Transcriptional Output in Gastrointestinal Stromal Tumor2022 · chromatin | 78 | 1 | 2.0 | APT 0.533 cites |
| GSE206257 | Concurrent inhibition of CDK2 adds to the anti-tumor activity of CDK4/6 inhibition in GIST2022 · sequencing | 12 | 1 | 1.8 | mixedAPT 0.527 cites |
| GSE225819 | Expression Profiling Identifies Key Genes in GIST Patients with Liver Metastasis2023 · array | 40 | 4 | 0.5 | patient cohortAPT 0.054 cites |
| GSE225978 | Chromosomal instability and deregulated cell cycle pathways are associated with increased metastatic capacity in treatment naïve, localized, high-risk gastrointestinal stromal tumours2023 · array | 21 | 1 | 0.1 | patient cohortAPT 0.05stagemolecularIMATINIB2 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-16. SubSeries are collapsed to one row per study by linked PMID. Of 165 series retrieved, 10 were dropped by the profile’s exclusion rules and 55 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.
SDHB
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
SDHA
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
SDHC
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
SDHD
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
NF1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
ETV1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
ANO1
1 approved drug (Crofelemer) and no registered trial in gastrointestinal stromal tumour. The molecules exist; nobody has tested them here.
CDKN2A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
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 (2026-09-16), ClinicalTrials.gov (2026-09-16), openFDA (2026-09-16), NCBI GEO (2026-09-16), PubMed (2026-09-16), NIH RePORTER (2026-09-16).
Negatives stated explicitly
Where nothing exists for gastrointestinal stromal tumour — no drug, no trial, no approval — the page says so in those words rather than omitting the row. An absent row is unquotable; a stated 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
5 exclusion patterns are applied to free text before anything is ranked, because none of note is used as a model system in gastrointestinal stromal tumour. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Gastrointestinal stromal tumour",
"mesh": "Gastrointestinal Stromal Tumors",
"facts": "https://usebiotransfer.org/disease/gastrointestinal-stromal-tumor.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."
}