Disease Briefing

Small intestine cancer: 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 2026-09-12Sources Open Targets · ClinicalTrials.gov · openFDA · GEO · PubMed · Europe PMC · iCite · RePORTERRanked 16 studies · 901 GEO samples
Reading as
Reorders emphasis only — nothing is hidden or loaded on demand.

Answer block

12 genes recurrently implicated in small intestine cancer — KRAS, TP53, APC, SMAD4, ERBB2, BRAF, KIT, PDGFRA, MEN1, CDKN1B, SSTR2, MTOR — 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 small intestine cancer: Avapritinib, Everolimus, Lanreotide, Lutetium Lu 177 Dotatate, Octreotide, Regorafenib 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 small intestine cancer itself — BRAF, KIT, PDGFRA, SSTR2. Across all of them 192 drug entries reach these genes, 176 distinct once salt forms are merged
2
registered PD-1 cell-therapy trials in small intestine cancer, 0 active. Counted from ClinicalTrials.gov across 3 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 have no clinical programme of any kind: APC, SMAD4, MEN1, CDKN1B. ERBB2 has cell-therapy trials, so it is undrugged rather than untouched
491
human GEO series match the disease; 16 survive on-topic filtering, and only 2 are patient cohorts of 100+ samples
11
Europe PMC full-text papers name GSE140312 — the highest accession-mention count in the set. A mention is not proof of reanalysis, so read it as reach rather than reuse
$2.3M
NIH obligations in FY2025, across 3 distinct core projects — and none in FY2013: no project matched the disease that year. There is no growth rate to give from a base of zero

Target landscape

Open Targets · retrieved 2026-09-12 · weekly

12 genes recurrently implicated in small intestine cancer, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.

Of 12 recurrently implicated genes, 9 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Small intestine cancer 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 small intestine cancer 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
KRAS across cancers → 3 2 approvedAdagrasib, SotorasibApproved in non-small cell lung carcinoma. No small intestine cancer indication appears on these drugs’ labels.2 active of 8 small intestine cancer trials antibody, 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
APC across cancers → 0 No drug antibody, protein degrader, small molecule
SMAD4 across cancers → 0 No drug protein degrader, small molecule
ERBB2 across cancers → 45 17 approvedAfatinib, Afatinib Dimaleate, Dacomitinib, Lapatinib, Lapatinib Ditosylate, Margetuximab, Masoprocol, Neratinib, Pertuzumab, Trastuzumab, Trastuzumab Deruxtecan, Trastuzumab Duocarmazine, Trastuzumab Emtansine, Tucatinib, Vandetanib, Zanidatamab, ZenocutuzumabApproved in non-small cell lung carcinoma, breast cancer, breast neoplasm and 10 other indications. No small intestine cancer indication appears on these drugs’ labels.10 active of 26 small intestine cancer trials antibody, other clinical modality, protein degrader, small molecule 0 active of 2 trials
BRAF across cancers → 16 6 approvedDabrafenib, Encorafenib, Regorafenib, Sorafenib, Tovorafenib, VemurafenibApproved in small intestine cancer: Regorafenib.5 active of 13 small intestine cancer trials antibody, protein degrader, small molecule
KIT across cancers → 33 15 approvedAvapritinib, Cediranib, Dasatinib, Imatinib, Masitinib, Midostaurin, Pazopanib, Pexidartinib, Quizartinib, Regorafenib, Ripretinib, Sorafenib, Sunitinib, Sunitinib Malate, TivozanibApproved in small intestine cancer: Avapritinib, Regorafenib, Ripretinib, Sunitinib.2 active of 21 small intestine cancer 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 small intestine cancer: Avapritinib, Regorafenib, Ripretinib, Sunitinib.2 active of 14 small intestine cancer trials antibody, other clinical modality, protein degrader, small molecule
MEN1 across cancers → 2 1 approvedRevumenibApproved in leukemia. No small intestine cancer indication appears on these drugs’ labels.No small intestine cancer trial of any of these drugs protein degrader, small molecule
CDKN1B 0 No drug protein degrader, small molecule
SSTR2 12 6 approvedLanreotide, Lutetium Oxodotreotide Lu-177, Octreotide, Paltusotine, Pasireotide, Pasireotide PamoateApproved in small intestine cancer: Lanreotide, Octreotide.12 trials, none active antibody, other clinical modality, protein degrader, small molecule
MTOR across cancers → 25 2 approvedPerhexiline, RidaforolimusApproved in cardiovascular disorder, angina pectoris. No small intestine cancer indication appears on these drugs’ labels.1 trials, none active antibody, protein degrader, small molecule

Dataset evidence counts studies in the 16-study ranked set whose title or abstract names the gene; the bar is scaled to APC. Sources: Open Targets Platform and NCBI GEO, retrieved 2026-09-12. 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 small intestine cancer: Avapritinib, Everolimus, Lanreotide, Lutetium Lu 177 Dotatate, Octreotide, Regorafenib, Ripretinib, Sunitinib. Separately, 45 of the drugs returned for the genes in the table above are approved only for other diseases and reach small intestine cancer through trials, not through their labels.

8Labelled for small intestine cancerFDA INDICATIONS AND USAGE names the disease
45Approved, but for another diseasereturned for the genes in the table above
0Backbone agents listing itbroad cytotoxics whose labels name many tumours
0Active PD-1 cell-therapy trialsof 2 registered

Every label that names small intestine cancer

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
EverolimusAfinitor, Afinitor Disperz, EverolimusLabelled hereLimitations of Use: AFINITOR is not indicated for the treatment of patients with functional carcinoid tumors.
LanreotideLANREOTIDE ACETATE, Lanreotide Acetate, SOMATULINE DEPOTLabelled hereGastroenteropancreatic Neuroendocrine Tumors SOMATULINE DEPOT is indicated for the treatment of adult patients with unresectable, well or moderately differentiated, locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors (GEP-NETs) to improve progression-free survival.
Lutetium Lu 177 DotatateLutatheraLabelled hereLUTATHERA is indicated for the treatment of adult and pediatric patients 12 years and older with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors.
OctreotideBYNFEZIA Pen, OCTREOTIDE ACETATE, OctreotideLabelled hereCarcinoid Tumors Long-term treatment of the severe diarrhea and flushing episodes associated with metastatic carcinoid tumors.
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.

14 labels match indications_and_usage:"small intestine cancer" OR indications_and_usage:"cancer of the small intestine" OR indications_and_usage:"small bowel adenocarcinoma" OR indications_and_usage:"small intestinal adenocarcinoma" OR indications_and_usage:"gastroenteropancreatic neuroendocrine" OR indications_and_usage:"midgut carcinoid" OR indications_and_usage:"carcinoid tumors" OR indications_and_usage:"gastrointestinal stromal tumor"; they collapse to 8 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Source: openFDA drug label API (api.fda.gov/drug/label.json), retrieved 2026-09-12. Labels change; this reflects the current label text, not the approval history. Not prescribing guidance.

Cell therapy against PD-1

ClinicalTrials.gov · retrieved 2026-09-12 · weekly

PD-1 is the busiest cell-therapy antigen in small intestine cancer: 2 registered trials, 0 still active. It has no gene entry of its own and is reached through PDCD1: a form or product of that gene.

TrialPhaseStatusTitleLast update
NCT039703821SuspendedA Study of Gene Edited Autologous Neoantigen Targeted TCR T Cells With or Without Anti-PD-1 in Patients With Solid Tumors2022-08-18
NCT029830451/2CompletedA Dose Escalation and Cohort Expansion Study of NKTR-214 in Combination With Nivolumab and Other Anti-Cancer Therapies in Patients With Select Advanced Solid Tumors2023-03-13

2 of 2 shown, most recently active first. Other antigens searched: HER2 (2), EGFR (2), VEGF (1). Source: ClinicalTrials.gov API v2, retrieved 2026-09-12. Trials are matched on the antigen named in the title or intervention, so a trial stating only a product code is missed.

Research momentum

PubMed MeSH · monthly

Every term below is a share of the small intestine cancer literature, not a count, because the field itself grew: 2015–2018 (n=1,775) against 2021–2025 (n=1,649). A topic whose papers doubled while the field doubled has not risen.

Rising, with a real baseline

MeSH term2015–182021–25ChangeShare now
Neoplasms, Glandular and Epithelial0.39%2.37%6.0×39 papers
Quality of Life0.28%0.91%3.23×15 papers
Robotic Surgical Procedures0.28%0.91%3.23×15 papers
Goblet Cells0.51%1.58%3.11×26 papers
Organometallic Compounds0.39%1.21%3.07×20 papers
Appendix3.55%8.91%2.51×147 papers
Neoplasms, Cystic, Mucinous, and Serous0.51%1.21%2.39×20 papers
Cytoreduction Surgical Procedures3.83%8.91%2.33×147 papers
Endoscopic Mucosal Resection3.49%7.52%2.15×124 papers
Propensity Score0.51%1.09%2.15×18 papers

Cooling

MeSH term2015–182021–25ChangeShare now
Antineoplastic Agents3.94%0.73%0.18×12 papers
Survival Analysis3.04%0.85%0.28×14 papers
Immunohistochemistry6.25%1.76%0.28×29 papers
Biopsy6.03%1.82%0.3×30 papers
Tumor Burden2.99%0.91%0.3×15 papers
Endoscopy, Gastrointestinal4.28%1.27%0.3×21 papers

Biggest topics now

MeSH term2015–182021–25ChangeShare now
Appendiceal Neoplasms19.89%35.66%1.79×588 papers
Duodenal Neoplasms22.82%28.08%1.23×463 papers
Adenocarcinoma16.56%18.5%1.12×305 papers
Neuroendocrine Tumors11.77%16.13%1.37×266 papers
Intestine, Small14.42%13.95%0.97×230 papers
Peritoneal Neoplasms7.38%12.31%1.67×203 papers
Prognosis10.87%12.07%1.11×199 papers
Treatment Outcome15.55%10.73%0.69×177 papers
Adenocarcinoma, Mucinous6.14%9.95%1.62×164 papers
Appendectomy5.92%9.88%1.67×163 papers

Publication mix

Type2015–182021–25
Randomized Controlled Trial0.5%0.2%
Review11.8%9.9%
Meta-Analysis0.6%0.8%
Case Reports0.0%9.2%

Query: Intestinal Neoplasms[MeSH Major Topic] NOT ("Colorectal Neoplasms"[MeSH] OR "Colonic Neoplasms"[MeSH] OR "Rectal Neoplasms"[MeSH] OR "Stomach Neoplasms"[MeSH] OR "Ampulla of Vater"[MeSH] OR "Common Bile Duct Neoplasms"[MeSH] OR "Pancreatic Neoplasms"[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 year14,450 cases/yeardirect2026
Deaths each year2,170 deaths/yeardirect2026
Incidence rate2.7 cases per 100,000 people per yeardirect2019-2023
Death rate0.5 deaths per 100,000 people per yeardirect2020-2024
People living with it93,952 people living with the diseasedirect2023
Median age at diagnosis66.0 yearsdirect2019-2023
median age at death73 yearsdirect2020-2024
Five-year relative survival71.8%direct2016-2022

Years of life lost

3.5 years per case, 50,529 a year, from survival and age at diagnosis rather than from a death count. derived proxy 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 small intestine cancer, after removing the 1,701 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.

$2.3MNIH obligations, FY2025from $0.2M in FY2016 · +1062%
3distinct projects funded1 in FY2016
$2.3Mpeak year was FY2025obligations, all institutes
33%of FY2025 awards from NIDDK1 of 3

NIH obligations by fiscal year

$1M$1M$2M$2M161820222425
YearObligationsAwardsDistinct projectsDropped as off-topic
FY2016$0.2M11110
FY2017$0.5M22113
FY2018$0.9M22134
FY2019$0.6M11120
FY2020$1.1M22126
FY2021$1.2M22123
FY2022$1.7M33170
FY2023$1.5M22155
FY2024$1.6M22158
FY2025$2.3M33137

Where FY2025 money went

InstitutionObligationsAwards
National Institute Of Diabetes And Digestive And Kidney Diseases$1.0M1
National Heart, Lung, And Blood Institute$0.7M1
Dana-Farber Cancer Inst$0.6M1

Text search small intestine cancer 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 2026-09-12.

What that buys

Against 50,529 years of life lost a year, FY2025 obligations are $45 per life-year — $158 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

NIDDK1 · 33%
NCI1 · 33%
NHLBI1 · 33%

Projects by administering institute. The rows above are the top 3 and account for 3 of 3.

Award mechanisms

ZIA2 · 67%
P011 · 33%

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 2 and account for 3 of 3.

Where it lands

National Institute Of Diabetes And Diges$1.0M · 44.5%
National Heart, Lung, And Blood Institut$0.7M · 29.7%
Dana-Farber Cancer Inst$0.6M · 25.8%

Share of $2.3M in FY2025. The top three hold 100%.

Public datasets, ranked

NCBI GEO + Europe PMC + iCite · weekly

491 human GEO series match small intestine cancer. Keyword relevance cannot tell a 133-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 16-study ranked set

patient 9unspecified 6mixed 1
9 patient6 unspecified1 mixed7 carry clinical annotation4 carry survival2 patient cohorts ≥100 GEO samples901 GEO samples totalin 2 single-cell series a GEO sample is one cell, not one patient

Established

high reuse, older
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE73832Integrated DNA methylation and RNA expression in Small Intestinal neuroendocrine tumors2016 · methylation13325.3
patient cohortAPT 0.75survivalmolecularCDKN1B145 cites
GSE33568MicroRNA expression profiling of small-intestine neuroendocrine tumors and human neuroendocrine tumor cell lines2013 · array2533.0
mixedAPT 0.75survival95 cites
GSE9576Gene expression profiling of classical midgut carcinoid primary tumors and liver metastasis2008 · array1262.8
patient cohortAPT 0.75120 cites
GSE153314SMAD4 haploinsufficiency in small intestinal neuroendocrine tumors2021 · array13111.0
patient cohortAPT 0.25molecularCDKN1BSMAD415 cites
GSE111156Gene Expression Changes in the Duodenal Adenoma-Carcinoma Sequence in Familial Adenomatous Polyposis (FAP)2018 · array485
patient cohortstagemolecularAPC
GSE140312Single-Cell RNA-Seq Analysis of Metastasis-Primary Tumor Dissimilarity in a Patient with Gastrointestinal Neuroendocrine Cancer2020 · single-cell2110.5
patient cohortAPT 0.2518 cites
GSE61467DNA methylation screening of small bowel adenocarcinoma2015 · methylation5641.2
patient cohortAPT 0.7541 cites
GSE23418Small bowel adenocarcinoma copy number profiles are more closely related to colorectal than to gastric cancers2011 · array9100.8
patient cohortAPT 0.7537 cites

Recent

2022 onward, by score
AccessionStudySamplesMentionsEurope PMCRCREvidence
GSE229203Origins, diversity and clinical relevance of small intestinal neuroendocrine tumors [methylation]2025 · methylation20025.2
APT 0.5survival17 cites
GSE279475Single-Cell RNA-seq analysis of small intestine neuroendocrine tumors reveals the cell of origin and trajectory of gene-expression associated with the early tumor development2025 · single-cell340
patient cohortAPT 0.053 cites
GSE277215Insights into intestinal neuroendocrine tumors in relation to normal human EEC differentiation2026 · chromatin531
APT 0.05
GSE216067Exposure to the olfactory receptor 51E1 agonist nonanoic acid alters small intestinal neuroendocrine tumor phenotype2022 · sequencing210.6
APT 0.257 cites
GSE301265Spatial Transcriptomics Reveals Local Subtype-Specific Identity and Signaling within Multifocal Small Intestinal Neuroendocrine Tumors2026 · spatial81
APT 0.05

Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. SubSeries are collapsed to one row per study by linked PMID. Of 491 series retrieved, 185 were dropped by the profile’s exclusion rules and 285 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.

APC

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

SMAD4

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

MEN1

1 approved drug (Revumenib) and no registered trial in small intestine cancer. The molecules exist; nobody has tested them here.

CDKN1B

No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.

SSTR2

16 registered trials, 7 withdrawn before enrolling anyone and none active. This target reads as tried; it was not.

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 (2026-09-12), ClinicalTrials.gov (2026-09-12), openFDA (2026-09-12), NCBI GEO (2026-09-12), PubMed (2026-09-12), NIH RePORTER (2026-09-12).

Negatives stated explicitly

Where nothing exists for small intestine cancer — 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

4 exclusion patterns are applied to free text before anything is ranked, because HCT-8 is used as a model system in Cryptosporidium and enteric-pathogen culture, intestinal epithelial infection models. What was removed and why is stated in each section rather than silently applied.

{
  "disease": "Small intestine cancer",
  "mesh": "Intestinal Neoplasms",
  "facts": "https://usebiotransfer.org/disease/small-intestine-cancer.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."
}