Disease Briefing

Gastrointestinal stromal tumour: 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 2026-09-16Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 76 studies · 2,509 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in gastrointestinal stromal tumour — KIT, PDGFRA, SDHB, SDHA, SDHC, SDHD, NF1, BRAF, ETV1, ANO1, CDKN2A, TP53 — 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.

6
drugs carry an FDA label naming gastrointestinal stromal tumour: Avapritinib, Imatinib, Pazopanib, Regorafenib, Ripretinib, Sunitinib. This counts labels, not treatment — the disease is also treated with agents approved under broader indications
3
of the 12 genes above carry a drug that is approved in gastrointestinal stromal tumour itself — BRAF, KIT, PDGFRA. Across all of them 99 drug entries reach these genes, 90 distinct once salt forms are merged
0
registered KIT cell-therapy trials in gastrointestinal stromal tumour, 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
8
targets carry an Open Targets tractability signal and have no clinical programme of any kind: SDHB, SDHA, SDHC, SDHD, NF1, ETV1, ANO1, TP53
165
human GEO series match the disease; 76 survive on-topic filtering, and only 3 are patient cohorts of 100+ samples
16
Europe PMC full-text papers name GSE8167 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$12.1M
NIH obligations in FY2025, up 435% since 2013 — while distinct core projects went 7 to 9. Larger awards rather than more distinct core projects; core projects are not the same unit as laboratories

Target landscape

Open Targets · retrieved 2026-09-16 · weekly

12 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.

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
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 · weekly

6 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.

6Labelled for gastrointestinal stromal tumourFDA INDICATIONS AND USAGE names the disease
18Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours

Every label that names gastrointestinal stromal tumour

DrugRoleWhat the label says
AvapritinibAyvakitLabelled herePDGFRA 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, IMKELDILabelled hereKit+ Gastrointestinal Stromal Tumors (GIST) Patients with Kit (CD117) positive unresectable and/or metastatic malignant gastrointestinal stromal tumors.
PazopanibPazopanib, VOTRIENT, pazopanibLabelled hereLimitations of Use : The efficacy of VOTRIENT for the treatment of patients with adipocytic STS or gastrointestinal stromal tumors has not been demonstrated.
RegorafenibStivargaLabelled hereGastrointestinal 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.
RipretinibQINLOCKLabelled hereQINLOCK 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 MalateLabelled hereGastrointestinal 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 · weekly

No 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 · monthly

Every 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 term2015–182021–25ChangeShare now
Soft Tissue Neoplasms0.36%1.51%4.17×24 papers
Propensity Score0.51%1.7%3.35×27 papers
Tumor Microenvironment0.36%1.2%3.3×19 papers
Progression-Free Survival0.58%1.64%2.82×26 papers
Nomograms0.65%1.77%2.7×28 papers
Quality of Life0.65%1.51%2.32×24 papers
Endoscopic Mucosal Resection1.74%3.91%2.24×62 papers
Rectum1.09%2.4%2.2×38 papers
Drug Monitoring0.44%0.88%2.03×14 papers
Neuroendocrine Tumors0.58%1.13%1.95×18 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Neoplasm Staging5.88%1.01%0.17×16 papers
Time Factors5.3%1.07%0.2×17 papers
Neoplasm Metastasis3.56%0.76%0.21×12 papers
Biopsy3.77%0.82%0.22×13 papers
Disease-Free Survival8.85%2.02%0.23×32 papers
Tumor Burden5.59%1.32%0.24×21 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Gastrointestinal Neoplasms36.07%27.3%0.76×433 papers
Imatinib Mesylate25.76%24.4%0.95×387 papers
Stomach Neoplasms20.39%23.2%1.14×368 papers
Antineoplastic Agents24.38%22.13%0.91×351 papers
Proto-Oncogene Proteins c-kit15.67%15.26%0.97×242 papers
Treatment Outcome20.46%14.19%0.69×225 papers
Mutation13.72%13.75%1.0×218 papers
Prognosis17.85%12.8%0.72×203 papers
Protein Kinase Inhibitors10.09%9.84%0.98×156 papers
Neoplasm Recurrence, Local12.26%8.64%0.7×137 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial1.5%1.6%
Review12.3%10.0%
Meta-Analysis1.5%1.2%
Case Reports0.0%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.

MeasureValueMatchWhat it counts
New cases each year6,000 cases/yeardirect
Deaths each yearnot publishedNot published on the page; SEER counts GIST deaths under the organ of origin.
Five-year relative survivalnot publishedThe 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 diagnosisnot publishedNot 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 · quarterly

NIH 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.

$12.1MNIH obligations, FY2025from $2.3M in FY2013 · +435%
9distinct projects funded7 in FY2013
$24.0Mpeak year was FY2021obligations, all institutes
100%of FY2025 awards from NCI9 of 9

NIH obligations by fiscal year

$6M$12M$18M$24M13151719212325
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2013$2.3M773
FY2014$1.7M883
FY2015$1.4M773
FY2016$1.7M663
FY2017$3.6M771
FY2018$3.3M771
FY2019$2.8M771
FY2020$8.8M11100
FY2021$24.0M770
FY2022$2.4M762
FY2023$7.2M662
FY2024$2.1M551
FY2025$12.1M990

Where FY2025 money went

InstitutionObligationsAwards
Division Of Basic Sciences - Nci$9.4M3
University Of California, San Diego$1.3M2
University Of Pennsylvania$0.5M2
Sloan-Kettering Inst Can Research$0.5M1
Research Inst Of Fox Chase Can Ctr$0.4M1

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

NCI9 · 100%

Projects by administering institute. The rows above are the top 1 and account for 9 of 9.

Award mechanisms

R013 · 33%
P501 · 11%
F321 · 11%
U011 · 11%
ZIA1 · 11%
ZIE1 · 11%

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

Division Of Basic Sciences - Nci$9.4M · 77.4%
University Of California, San Diego$1.3M · 10.7%
University Of Pennsylvania$0.5M · 4.3%
Sloan-Kettering Inst Can Research$0.5M · 4.0%
Research Inst Of Fox Chase Can Ctr$0.4M · 3.5%

Share of $12.1M in FY2025. The top three hold 92%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

165 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

unspecified 41patient 20cell line 9mixed 6
41 unspecified20 patient9 cell line6 mixed50 carry clinical annotation12 carry survival3 patient cohorts ≥100 GEO samples2,509 GEO samples totalin 3 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE21123Affymetrix SNP array data for soft tissue sarcoma samples2010 · array415112.9
patient cohortAPT 0.95survivalmolecularKITNF1TP53614 cites
GSE107447Altered chromosomal topology drives oncogenic programs in SDH-deficient GISTs2019 · chromatin10056.0
patient cohortAPT 0.75molecularKITPDGFRA210 cites
GSE136755Driver gene alterations and activated signaling pathways toward malignant progression of gastrointestinal stromal tumors2019 · array65151.1
patient cohortAPT 0.5stagemolecularKITPDGFRA28 cites
GSE56670Expression data from SDH-disabled GIST2015 · array2054.4
patient cohortAPT 0.95molecularSDHASDHBSDHC157 cites
GSE34387Oncogenic SDH mutation underlies global epigenomic instability in gastrointestinal stromal tumor2013 · methylation22036.9
APT 0.75molecularKITPDGFRA279 cites
GSE8167Distinct gene-expression-defined classes of gastrointestinal stromal tumor (GIST).2008 · array32162.1
APT 0.75molecularKITPDGFRA97 cites
GSE15966Gene expression signature predictive of rapid response to imatinib mesylate in Gastrointestinal Stromal Tumor (GIST)2009 · array5471.0
patient cohortAPT 0.5molecularIMATINIB44 cites
GSE155800Identification of transcriptional difference between Imatinib-sensitive and resistant patients with gastrointerstinal stromal tumor (GIST)2021 · sequencing2090.8
patient cohortAPT 0.25molecularIMATINIB14 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE254762Deciphering the Tumor Immune Microenvironment of Imatinib-Resistance in Advanced Gastrointestinal Stromal Tumors at Single-Cell Resolution2024 · single-cell953.1
patient cohortAPT 0.5molecularIMATINIB26 cites
GSE151323Anatomic and transcriptional determinants of gastrointestinal stromal tumor2022 · sequencing911.4
APT 0.5survivalmolecularIMATINIBKIT24 cites
GSE172154MOZ and Menin-MLL Complexes are Complementary Regulators of Chromatin Association and Transcriptional Output in Gastrointestinal Stromal Tumor2022 · chromatin7812.0
APT 0.533 cites
GSE206257Concurrent inhibition of CDK2 adds to the anti-tumor activity of CDK4/6 inhibition in GIST2022 · sequencing1211.8
mixedAPT 0.527 cites
GSE225819Expression Profiling Identifies Key Genes in GIST Patients with Liver Metastasis2023 · array4040.5
patient cohortAPT 0.054 cites
GSE225978Chromosomal instability and deregulated cell cycle pathways are associated with increased metastatic capacity in treatment naïve, localized, high-risk gastrointestinal stromal tumours2023 · array2110.1
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 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.

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

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 (2026-09-16), 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."
}