Target landscape
Open Targets · retrieved 2026-09-12 · weekly12 genes recurrently implicated in cholangiocarcinoma, triaged for whether anything can actually be aimed at them. The zeros are the informative rows.
Of 12 recurrently implicated genes, 7 carry any drug at all. That is a statement about these 12 gene targets, not about the disease: Cholangiocarcinoma does have labelled therapy — 7 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 cholangiocarcinoma 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 |
|---|---|---|---|---|
| FGFR2 across cancers → | 26 | 10 approvedE-7090, Erdafitinib, Futibatinib, Infigratinib, Nintedanib, Nintedanib Esylate, Palifermin, Pemigatinib, Regorafenib, TraferminApproved in cholangiocarcinoma: Futibatinib, Pemigatinib.13 active of 38 cholangiocarcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
| IDH1 across cancers → | 7 | 3 approvedIvosidenib, Olutasidenib, VorasidenibApproved in cholangiocarcinoma: Ivosidenib.7 active of 12 cholangiocarcinoma trials | antibody, protein degrader, small molecule | — |
| KRAS across cancers → | 3 | 2 approvedAdagrasib, SotorasibApproved in non-small cell lung carcinoma. No cholangiocarcinoma indication appears on these drugs’ labels.No cholangiocarcinoma trial of any of these drugs | antibody, protein degrader, small molecule | — |
| TP53 across cancers → | 8 | Phase 3Alrizomadlin, Cenersen, Contusugene Ladenovec, Eprenetapopt, Idasanutlin, Navtemadlin, Siremadlin, TeprasiranNo cholangiocarcinoma trial of any of these drugs | other clinical modality, 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 cholangiocarcinoma: Zanidatamab, Zenocutuzumab.10 active of 33 cholangiocarcinoma trials | antibody, other clinical modality, protein degrader, small molecule | 1 active of 1 trial |
| BRAF across cancers → | 16 | 6 approvedDabrafenib, Encorafenib, Regorafenib, Sorafenib, Tovorafenib, VemurafenibApproved in metastatic melanoma, low grade glioma, colorectal neoplasm and 4 other indications. No cholangiocarcinoma indication appears on these drugs’ labels.2 active of 13 cholangiocarcinoma trials | antibody, protein degrader, small molecule | — |
| BAP1 across cancers → | 0 | No drug— | protein degrader | — |
| ARID1A across cancers → | 0 | No drug— | protein degrader | — |
| CDKN2A across cancers → | 0 | No drug— | — | — |
| PBRM1 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| SMAD4 across cancers → | 0 | No drug— | protein degrader, small molecule | — |
| CD274 across cancers → | 13 | 5 approvedAtezolizumab, Avelumab, Cosibelimab, Durvalumab, SugemalimabApproved in cholangiocarcinoma: Durvalumab.50 active of 74 cholangiocarcinoma trials | antibody, other clinical modality, protein degrader, small molecule | — |
Dataset evidence counts studies in the 192-study ranked set whose title or abstract names the gene; the bar is scaled to KRAS. 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 · weekly7 drugs carry an FDA label naming cholangiocarcinoma: Durvalumab, Futibatinib, Ivosidenib, Pembrolizumab, Pemigatinib, Zanidatamab, Zenocutuzumab. Separately, 36 of the drugs returned for the genes in the table above are approved only for other diseases and reach cholangiocarcinoma through trials, not through their labels.
Every label that names cholangiocarcinoma
| Drug | Role | What the label says |
|---|---|---|
| DurvalumabIMFINZI | Labelled here | Biliary Tract Cancers IMFINZI, in combination with gemcitabine and cisplatin, is indicated for the treatment of adult patients with locally advanced or metastatic biliary tract cancer (BTC). |
| FutibatinibLYTGOBI | Labelled here | LYTGOBI is a kinase inhibitor indicated for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. |
| IvosidenibTIBSOVO | Labelled here | Locally Advanced or Metastatic Cholangiocarcinoma For the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated |
| PembrolizumabKEYTRUDA, KEYTRUDA QLEX | Labelled here | Biliary Tract Cancer (BTC) in combination with gemcitabine and cisplatin, for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer. |
| PemigatinibPEMAZYRE | Labelled here | Cholangiocarcinoma PEMAZYRE is indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test [see Dosage and Administration |
| ZanidatamabZIIHERA | Labelled here | Biliary Tract Cancer (BTC) • for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) BTC, as detected by an FDA-authorized test.* |
| ZenocutuzumabBIZENGRI | Labelled here | Adults with advanced, unresectable or metastatic cholangiocarcinoma harboring a neuregulin 1 ( NRG1 ) gene fusion with disease progression on or after prior systemic therapy. |
| Indocyanine Green And WaterIndocyanine Green | Not a therapy | Visual assessment of the major extrahepatic bile duct For visual assessment of the major extrahepatic bile ducts with OLYMPUS infrared imaging endoscopic imaging system. |
6 labels match indications_and_usage:"cholangiocarcinoma" OR indications_and_usage:"biliary tract cancer" OR indications_and_usage:"bile duct"; they collapse to 8 distinct molecules once salt forms, biosimilars and co-formulated hyaluronidase are merged. Diagnostic and contrast agents (Indocyanine Green And Water) are listed but are not treatments. 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 · weeklyPD-1 is the busiest cell-therapy antigen in cholangiocarcinoma: 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.
| Trial | Phase | Status | Title | Last update |
|---|---|---|---|---|
| NCT02757391 | 1 | Terminated | CD8+ T Cell Therapy and Pembrolizumab in Treating Patients With Metastatic Gastrointestinal Tumors | 2020-10-30 |
| NCT03937895 | 1/2 | Completed | Allogeneic NK Cell ("SMT-NK") in Combination With Pembrolizumab in Advanced Biliary Tract Cancer | 2022-03-29 |
2 of 2 shown, most recently active first. Other antigens searched: HER2 (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 · monthlyEvery term below is a share of the cholangiocarcinoma literature, not a count, because the field itself grew: 2015–2018 (n=1,977) against 2021–2025 (n=3,717). 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 |
|---|---|---|---|---|
| Tumor Microenvironment | 1.11% | 7.1% | 6.38× | 264 papers |
| Immunotherapy | 0.66% | 2.45% | 3.72× | 91 papers |
| B7-H1 Antigen | 0.46% | 1.67% | 3.66× | 62 papers |
| Antibodies, Monoclonal, Humanized | 0.3% | 1.1% | 3.63× | 41 papers |
| Precision Medicine | 0.35% | 1.26% | 3.57× | 47 papers |
| Programmed Cell Death 1 Receptor | 0.25% | 0.89% | 3.51× | 33 papers |
| CD8-Positive T-Lymphocytes | 0.3% | 1.05% | 3.46× | 39 papers |
| Lymphocytes, Tumor-Infiltrating | 0.3% | 1.02% | 3.37× | 38 papers |
| Pyrimidines | 0.61% | 1.96% | 3.24× | 73 papers |
| Phosphatidylinositol 3-Kinases | 0.51% | 1.61% | 3.19× | 60 papers |
Cooling
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Time Factors | 6.27% | 0.91% | 0.15× | 34 papers |
| Survival Analysis | 5.36% | 0.83% | 0.16× | 31 papers |
| Proportional Hazards Models | 5.67% | 1.05% | 0.19× | 39 papers |
| Immunohistochemistry | 5.87% | 1.13% | 0.19× | 42 papers |
| Case-Control Studies | 2.83% | 0.54% | 0.19× | 20 papers |
| Japan | 1.72% | 0.35% | 0.2× | 13 papers |
Biggest topics now
| MeSH term | 2015–18 | 2021–25 | Change | Share now |
|---|---|---|---|---|
| Bile Duct Neoplasms | 87.25% | 94.78% | 1.09× | 3523 papers |
| Bile Ducts, Intrahepatic | 28.78% | 48.91% | 1.7× | 1818 papers |
| Prognosis | 23.22% | 22.73% | 0.98× | 845 papers |
| Hepatectomy | 16.74% | 14.61% | 0.87× | 543 papers |
| Klatskin Tumor | 14.21% | 13.29% | 0.94× | 494 papers |
| Cell Proliferation | 12.44% | 11.54% | 0.93× | 429 papers |
| Treatment Outcome | 16.34% | 9.82% | 0.6× | 365 papers |
| Gene Expression Regulation, Neoplastic | 10.07% | 8.66% | 0.86× | 322 papers |
| Biomarkers, Tumor | 12.7% | 7.94% | 0.63× | 295 papers |
| Tumor Microenvironment | 1.11% | 7.1% | 6.38× | 264 papers |
Publication mix
| Type | 2015–18 | 2021–25 |
|---|---|---|
| Clinical Trial | 0.1% | 0.0% |
| Randomized Controlled Trial | 0.6% | 0.6% |
| Review | 11.2% | 9.8% |
| Meta-Analysis | 2.3% | 1.9% |
| Case Reports | 0.0% | 2.9% |
Query: Cholangiocarcinoma[MeSH Major Topic] NOT ("Carcinoma, Hepatocellular"[MeSH] OR "Gallbladder Neoplasms"[MeSH] OR "Pancreatic Neoplasms"[MeSH] OR "Common Bile Duct 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.
| Measure | Value | Match | What it counts |
|---|---|---|---|
| New cases each year | 8,000 cases/year | direct | American Cancer Society, Key Statistics for Bile Duct Cancer, retrieved 2026-09-12 |
| New cases each year | 42,340 cases/year | proxy | counts liver and intrahepatic bile duct cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Liver and Intrahepatic Bile Duct Cancer, retrieved 2026-09-12 |
| Deaths each year | 30,980 deaths/year | proxy | counts liver and intrahepatic bile duct cancer, which is broader than this disease; 2026 SEER Cancer Stat Facts, Liver and Intrahepatic Bile Duct Cancer, retrieved 2026-09-12 |
| Incidence rate | 9.5 cases per 100,000 per year | proxy | counts liver and intrahepatic bile duct cancer, which is broader than this disease; 2019-2023 SEER Cancer Stat Facts, Liver and Intrahepatic Bile Duct Cancer, retrieved 2026-09-12 |
| Death rate | 6.6 deaths per 100,000 per year | proxy | counts liver and intrahepatic bile duct cancer, which is broader than this disease; 2020-2024 SEER Cancer Stat Facts, Liver and Intrahepatic Bile Duct Cancer, retrieved 2026-09-12 |
| People living with it | 116,514 people living with the disease | proxy | counts liver and intrahepatic bile duct cancer, which is broader than this disease; 2023 SEER Cancer Stat Facts, Liver and Intrahepatic Bile Duct Cancer, retrieved 2026-09-12 |
| Deaths each year | — | not published | Not published for the disease alone; the liver and intrahepatic bile duct figure is mostly hepatocellular carcinoma. |
| Five-year relative survival | — | not published | The American Cancer Society publishes five-year relative survival by stage and by site (intrahepatic, extrahepatic), not one figure; SEER has no page. An average across them would be a derivation the sources do not make. |
| Median age at diagnosis | — | not published | The American Cancer Society gives "in the 70s"; no median is published. |
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 cholangiocarcinoma, after removing the 513 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 | $3.9M | 14 | 13 | 15 |
| FY2014 | $2.6M | 11 | 9 | 19 |
| FY2015 | $2.9M | 11 | 10 | 21 |
| FY2016 | $3.1M | 12 | 11 | 24 |
| FY2017 | $2.2M | 12 | 11 | 23 |
| FY2018 | $4.5M | 13 | 12 | 36 |
| FY2019 | $4.5M | 18 | 13 | 48 |
| FY2020 | $4.8M | 14 | 13 | 40 |
| FY2021 | $5.3M | 13 | 12 | 54 |
| FY2022 | $4.8M | 15 | 14 | 61 |
| FY2023 | $5.6M | 15 | 14 | 60 |
| FY2024 | $5.9M | 16 | 15 | 54 |
| FY2025 | $7.4M | 20 | 17 | 58 |
Where FY2025 money went
| Institution | Obligations | Awards |
|---|---|---|
| Mayo Clinic Rochester | $3.0M | 9 |
| University Of Pittsburgh At Pittsburgh | $0.8M | 2 |
| Sanford Burnham Prebys Medical Discovery Institute | $0.7M | 1 |
| Medical University Of South Carolina | $0.7M | 1 |
| Massachusetts General Hospital | $0.7M | 1 |
| Icahn School Of Medicine At Mount Sinai | $0.6M | 1 |
| Washington University | $0.5M | 1 |
| Fred Hutchinson Cancer Center | $0.4M | 1 |
| University Of Pennsylvania | $0.0M | 1 |
| Vanderbilt University | $0.0M | 1 |
Text search cholangiocarcinoma 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.
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 3 and account for 20 of 20.
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 8 and account for 20 of 20.
Where it lands
Share of $7.4M in FY2025. The top three hold 61%.
Public datasets, ranked
NCBI GEO + Europe PMC + iCite · weekly406 human GEO series match cholangiocarcinoma. Keyword relevance cannot tell a 1,403-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 192-study ranked set
Established
high reuse, older| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE26566 | Molecular classification of human cholangiocarcinoma2012 · array | 169 | 175 | 11.7 | patient cohortAPT 0.95survivalstagemolecularBRAFHER2KRAS475 cites |
| GSE76311 | Human hepatocellular carcinoma (HCC) and Cholangiocarcinoma (CCA) from Thailand Initiative in Genomics and Expression Research for Liver Cancer (TIGER-LC)2017 · array | 608 | 175 | 9.9 | APT 0.95351 cites |
| GSE33327 | Integrative Molecular Analysis of Intrahepatic Cholangiocarcinoma Reveals 2 Classes That Have Different Outcomes2013 · array | 283 | 63 | 12.0 | patient cohortAPT 0.95survivalmolecularBRAFKRAS456 cites |
| GSE132305 | Molecular classification and therapeutic targets in extrahepatic cholangiocarcinoma2020 · array | 220 | 49 | 11.2 | patient cohortAPT 0.95271 cites |
| GSE89749 | Integrative genomic and epigenetic analysis in cholangiocarcinoma2017 · array | 120 | 76 | 24.3 | APT 0.95777 cites |
| GSE59856 | MicroRNA markers for the diagnosis of pancreatic and biliary tract cancers2015 · array | 571 | 46 | 3.9 | patient cohortAPT 0.75113 cites |
| GSE107943 | Integrative molecular and clinical analysis of intrahepatic cholangiocarcinoma reveals two prognostic subclassees2018 · sequencing | 57 | 192 | 2.2 | APT 0.5survival64 cites |
| GSE49656 | Distinct Mutational Patterns of Infection and Non-Infection-Related Bile Duct Cancers Revealed by Exome Sequencing2013 · methylation | 40 | 15 | 10.6 | APT 0.95molecularARID1ABAP1IDH1419 cites |
Recent
2022 onward, by score| Accession | Study | Samples | MentionsEurope PMC | RCR | Evidence |
|---|---|---|---|---|---|
| GSE179443 | Molecular and radiopathologic spectrum between HCC and intrahepatic cholangiocarcinoma2022 · sequencing | 137 | 4 | 3.9 | patient cohortAPT 0.5survivalmolecularKRAS39 cites |
| GSE244807 | Self-supervised learning for predicting transcriptomic groups on whole slides images in intrahepatic cholangiocarcinoma2023 · sequencing | 246 | 11 | 0.8 | patient cohortAPT 0.5survival7 cites |
| GSE255058 | Clinical and biomarker analyses of hepatic arterial infusion chemotherapy plus lenvatinib and PD-1 inhibitor for patients with advanced intrahepatic cholangiocarcinoma2024 · sequencing | 18 | 2 | 2.5 | patient cohortAPT 0.95survivalmolecularPD-118 cites |
| GSE201241 | Integrative Analysis Defines Distinct Prognostic Subgroups of Intrahepatic Cholangiocarcinoma2022 · methylation | 64 | 12 | 3.1 | APT 0.75survival88 cites |
| GSE201425 | Single-cell atlas of diverse immune populations in the advanced biliary tract cancer microenvironment2022 · single-cell | 32 | 14 | 1.2 | cell lineAPT 0.524 cites |
| GSE210067 | Circulating monocytes associated with anti-PD-1 resistance in human biliary cancer induce T cell paralysis2022 · sequencing | 27 | 3 | 2.2 | APT 0.75molecularPD-141 cites |
Sources: NCBI GEO + Europe PMC + iCite, retrieved 2026-09-12. SubSeries are collapsed to one row per study by linked PMID. Of 406 series retrieved, 45 were dropped by the profile’s exclusion rules and 117 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.
KRAS
2 approved drugs (Adagrasib, Sotorasib) and no registered trial in cholangiocarcinoma. The molecules exist; nobody has tested them here.
BAP1
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
ARID1A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
CDKN2A
No drug, no trial, no clinical programme of any kind. Whatever the biology says, nothing has been built.
PBRM1
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.
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 (2026-09-12), 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 cholangiocarcinoma — 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 RBE is used as a model system in none of consequence for the line; "RBE" the term is radiobiology. What was removed and why is stated in each section rather than silently applied.
{
"disease": "Cholangiocarcinoma",
"mesh": "Cholangiocarcinoma",
"facts": "https://usebiotransfer.org/disease/cholangiocarcinoma.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."
}