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🗺️ Disease Atlas · Hepatobiliary disorder

cholangiocarcinoma

A carcinoma that arises from the intrahepatic biliary tree (intrahepatic cholangiocarcinoma) or from the junction, or adjacent to the junction, of the right and left hepatic ducts (hilar cholangiocarcinoma). Grossly, the malignant lesions are solid, nodular, and grayish. Morphologically, the vast majority of cases are adenocarcinomas. Signs and symptoms include malaise, weight loss, right upper quadrant abdominal pa…

🔔 Follow weekly
🧬8830genes / proteins with evidence
💊144drugs and clinical candidates
🧪12+recruiting trials worldwide
✅5approved
📈14phase 3
🧬

Understand cholangiocarcinoma

Cancer · Cells that grow out of control
🎓 Learn more

What it is

Bile duct cancer is rare. It can happen in the parts of the bile ducts that are outside or inside the liver. Cancer of the bile duct outside of the liver is much more common. Risk factors include having inflammation of the bile duct, ulcerative colitis, and some liver diseases.

Symptoms can include:

Jaundice

From MedlinePlus: Bile Duct Cancer (US National Library of Medicine) ↗

🧬 Cancer: Cancer is a group of diseases in which some of the body's cells grow and divide without control, and can spread to other parts of the body.

❓ Why it happens

  • Changes (mutations) build up in the genes that tell a cell when to grow, divide and die.
  • Most of these changes happen during life: tobacco, too much sun, alcohol, some infections (such as HPV and hepatitis B and C), radiation, obesity and ageing all raise the risk.
  • Some people inherit a faulty gene that raises their risk, such as BRCA1 or BRCA2.

🫀 What it does to the body

  • Cancer cells form a lump (tumour) that can press on or invade nearby organs.
  • They can travel through the blood or lymph to start new tumours elsewhere (metastasis).
  • Blood cancers crowd out healthy blood cells in the bone marrow.

🧱 Why it is hard to cure

  • Every cancer is different, even between two people with the same type - and inside one tumour.
  • Cancer cells keep changing, so they can learn to resist a medicine that worked at first.
  • Cancer cells are the body's own cells, so killing them without harming healthy cells is hard.
  • Many cancers cause no symptoms until they have spread, and a new medicine often takes more than ten years to go from the lab to patients.

🚀 What research is trying now

  • Targeted medicines against the exact gene change in a tumour (for example EGFR, ALK or KRAS).
  • Immunotherapy that helps the immune system recognise cancer, including CAR-T cell therapy.
  • Personalised cancer vaccines built from each tumour's mutations, now in late trials.
  • Blood tests that look for tumour DNA to find cancer earlier, and AI to read scans and slides.

General information about cancer, reviewed by people; it does not describe any one person. Below: the evidence for cholangiocarcinoma itself.

🤖

AI briefing

Where the science on cholangiocarcinoma stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens cholangiocarcinoma.

🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
FGFR2 fibroblast growth factor receptor 2PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.67
IDH1 isocitrate dehydrogenase (NADP(+)) 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.65
FGFR4 fibroblast growth factor receptor 4PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.59
FGFR3 fibroblast growth factor receptor 3PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.59
FGFR1 fibroblast growth factor receptor 1PROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.59
TP53 tumor protein p53PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.55
KRAS KRas proto-oncogene, GTPasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.54
ARID1A AT-rich interaction domain 1APROTAC🧊 AlphaFold structure ↗ 0.53
BAP1 BRCA1 associated deubiquitinase 1PROTAC🧊 AlphaFold structure ↗ 0.50
PBRM1 polybromo 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.50
SMAD4 SMAD family member 4PROTACsmall molecule🧊 AlphaFold structure ↗ 0.49
CDKN2A cyclin dependent kinase inhibitor 2A🧊 AlphaFold structure ↗ 0.48
PIK3CA phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alphaPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.48
EPHA2 EPH receptor A2PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.45
BRAF B-Raf proto-oncogene, serine/threonine kinasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.44

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

💊

Drugs and candidates

Every medicine linked to cholangiocarcinoma, by how far it has got
Approved · 5
FutibatinibFibroblast growth factor receptor inhibitorApproved
InfigratinibFibroblast growth factor receptor inhibitorApproved
Infigratinib PhosphateFibroblast growth factor receptor inhibitorApproved
IvosidenibIsocitrate dehydrogenase [NADP] cytoplasmic inhibitorApproved
PemigatinibFibroblast growth factor receptor 2 inhibitor; Fibroblast growth factor receptor 3 inhibi…Approved
In phase 2 / 3 · 20
CapecitabineThymidylate synthase inhibitorPhase 3
CisplatinPhase 3
FluorouracilThymidylate synthase inhibitorPhase 3
GemcitabineRibonucleoside-diphosphate reductase RR1 inhibitor; DNA polymerase (alpha/delta/epsilon) …Phase 3
Gemcitabine HydrochlorideRibonucleoside-diphosphate reductase RR1 inhibitor; DNA polymerase (alpha/delta/epsilon) …Phase 3
LenvatinibVascular endothelial growth factor receptor inhibitorPhase 3
Show all 20
LeucovorinPhase 3
OxaliplatinPhase 3
PaclitaxelTubulin inhibitorPhase 3
Porfimer SodiumPhase 3
S1?Phase 3
TinengotinibPhase 3
TislelizumabProgrammed cell death protein 1 inhibitorPhase 3
TrastuzumabReceptor protein-tyrosine kinase erbB-2 inhibitorPhase 3
ToripalimabProgrammed cell death protein 1 antagonistPhase 2/3
Abc-294640Sphingosine kinase 2 inhibitorPhase 2
AbemaciclibCyclin-dependent kinase 4 inhibitor; Cyclin-dependent kinase 6 inhibitorPhase 2
AdebrelimabProgrammed cell death 1 ligand 1 inhibitorPhase 2
AfatinibEpidermal growth factor receptor erbB1 inhibitor; Receptor protein-tyrosine kinase erbB-2…Phase 2
AldesleukinInterleukin-2 receptor agonistPhase 2
Early trials · 20
AvelumabProgrammed cell death 1 ligand 1 otherPhase 1/2
BafisontamabHepatocyte growth factor receptor inhibitor; Epidermal growth factor receptor erbB1 inhib…Phase 1/2
BexmarilimabPhase 1/2
BirinapantcIAP1/cIAP2 inhibitorPhase 1/2
CyclophosphamidePhase 1/2
Dkn-01Dickkopf-related protein 1 inhibitorPhase 1/2
Show all 20
EzurpimtrostatPhase 1/2
FludarabinePhase 1/2
Hmpl-453Fibroblast growth factor receptor 1 inhibitor; Fibroblast growth factor receptor 2 inhibi…Phase 1/2
LirafugratinibPhase 1/2
NedisertibPhase 1/2
Octanoic AcidPhase 1/2
PalbociclibCDK6/cyclin D1 inhibitor; Cyclin-dependent kinase 4/cyclin D1 inhibitorPhase 1/2
Pasifolate ExatecanPhase 1/2
PenpulimabProgrammed cell death protein 1 inhibitorPhase 1/2
SilmitasertibCasein kinase II alpha inhibitorPhase 1/2
TivozanibVascular endothelial growth factor receptor inhibitor; Stem cell growth factor receptor i…Phase 1/2
Anetumab RavtansineMesothelin binding agent; Tubulin inhibitorPhase 1
CrelosidenibPhase 1
Fosgemcitabine PalabenamidePhase 1
🧪

Recruiting trials

Worldwide, open to new patients now
Adjuvant Therapy for High-Risk Intrahepatic Cholangiocarcinoma: A Real-World StudyNCT07640048 · n/a · 📍 BeijingTrop2-targeted immunoPET Imaging of Solid TumorsNCT06851663 · Phase 2, Phase 3 · 📍 ShanghaiPhase I/II Trial of Pemigatinib in Combination With Atezolizumab and Bevacizumab for Treatment of Advanced Ch…NCT06439485 · Phase 1, Phase 2 · 📍 HoustonGemcitabine and Oxaliplatin Chemotherapy With or Without a Floxuridine and Dexamethasone Pump in People With …NCT04891289 · Phase 2 · 📍 Bethesda68Ga-SorB PET/CT Imaging in Patients With Solid TumorsNCT07760012 · NA · 📍 BeijingREGULUS: MRI-guided Adaptive SABR for Liver CancersNCT07223307 · Phase 2 · 📍 Palo AltoPhase I/II Study: Allogeneic NK-cell Therapy With Chemotherapy for Post-Surgery PDA or Cholangiocarcinoma Pat…NCT06730009 · Phase 1, Phase 2 · 📍 TainanAlternating Chemo-immunotherapy and FGFR Inhibitor for FGFR-positive Biliary or CholangiocarcinomaNCT07780838 · Phase 2 · 📍 ColumbusEvaluation of diagnostiC Capacity of eccDNAs as Biomarkers in Indetermined biLiary Stricture(ECCBILE)NCT06277531 · n/a · 📍 BeijingA Study of Lirafugratinib in Non-CCA Solid Tumors With FGFR2 Fusion or RearrangementNCT07359820 · Phase 2 · 📍 PhoenixA Simple Breath Test to Detect Liver Cancer and Monitor Liver ConditionsNCT06864195 · n/a · 📍 LondonModified Immune Cells (Autologous Dendritic Cells) and a Vaccine (Prevnar) Combined With Immune Checkpoint In…NCT03942328 · Phase 1, Phase 2 · 📍 Rochester Search more trials →
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