DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for intrahepatic cholangiocarcinoma — screening already-approved drugs against its 41-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleIntrahepatic cholangiocarcinoma maps to a 41-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for intrahepatic cholangiocarcinoma is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
fibroblast growth factor receptor 4 (FGFR4) — FGFR4 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet 1~{r}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8KH8 · 1.49 Å · ligand 1-[4-[(1~{R})-1-[3,5-bis(chloranyl)pyridin-4-yl]ethoxy]-5-cyano-pyridin-2-yl]-3-[6-methanoyl-5-[(4-methyl-2-oxidanylidene-piperazin-1-yl)methyl]-3-(2-morpholin-4-ylethoxy)pyridin-2-yl]urea (VVW). Experimental structure, not a prediction.
What the evidence adds up to
Intrahepatic cholangiocarcinoma is a rare and aggressive malignancy with a five-year overall survival below 5% even among patients who undergo surgery. Resection is the only treatment considered curative, but only about 30% of patients present at a stage where surgery is possible, and intrahepatic recurrence is common even after complete resection. Conventional chemotherapy and radiotherapy are not effective for long-term survival and the overall prognosis remains poor.
Transarterial therapies have been discussed as a treatment option for intrahepatic cholangiocarcinoma, but the 2013 review does not report response rates or survival data from controlled trials. No evidence from that review shows that these procedures improve overall survival compared to supportive care alone.
Recent reviews from 2019 and 2021 note that no effective adjuvant treatment currently exists. Research has identified potential therapeutic targets in the tumour microenvironment, genes, proteins, epigenetic modifications, and signalling pathways, but these remain at the stage of preclinical or early clinical investigation. No targeted therapy has been shown in these abstracts to improve survival in a randomised trial.
What is still missing is adequate funding for large, randomised controlled trials that test these potential targets in well-stratified patient populations. Without such trials, and without a reliable way to identify which patients might benefit from which target, the gap between molecular discovery and any measurable improvement in survival remains unclosed.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Clinical Nuclear Medicine · 2015 · 64 citations
Glass Microspheres 90Y Selective Internal Radiation Therapy and Chemotherapy as First-Line Treatment of Intrahepatic Cholangiocarcinoma
AbstractPURPOSE OF THE REPORT: Intrahepatic cholangiocarcinoma's incidence is increasing. We studied the efficacy of Y selective internal radiation therapy (SIRT) as first-line treatment, with chemotherapy, and compared with the results of chemotherapy alone. PATIENTS AND METHODS: We retrospectively studied data from patients treated at our institution with glass microspheres SIRT for intrahepatic cholangiocarcinoma as part of first-line treatment in combination with chemotherapy. We compared results with those of similar patients treated in the ABC-02 study (a study in advanced biliary tract cancer that defined the current standard chemotherapy), assessed as not progressing after the first evaluation. We assessed progression-free survival (PFS) and overall survival (OS). RESULTS: Twenty-four patients were treated with SIRT. Chemotherapy was given concomitantly in 10 (42%), as induction before SIRT in 13 (54%) or after SIRT in 1 (4%). Grade 3 adverse events were reported in 1 (4%). Median PFS after SIRT was 10.3 months. Longer PFS was observed when chemotherapy was given concomitantly than when chemotherapy was given before SIRT, with respective median of 20.0 versus 8.8 months (P = 0.001). Median OS after SIRT was not reached. Eleven patients went to surgery (46%). Thirty-three patients in ABC-02 had locally advanced nonextrahepatic cholangiocarcinoma, not progressing after first evaluation. From the start of any treatment, the median PFS was 16.0 months in our cohort versus 11.3 months in ABC-02 (P = 0.25), whereas the median OS was significantly higher in our cohort, not reached versus 17.9 months, respectively (P = 0.026). CONCLUSIONS: Selective internal radiation therapy combined with concomitant chemotherapy seems a promising strategy as first-line treatment for unresectable intrahepatic cholangiocarcinoma.
Seminars in Interventional Radiology · 2013 · 19 citations · open access
Transarterial Therapies for the Treatment of Intrahepatic Cholangiocarcinoma
AbstractCholangiocarcinoma, whether arising from the intrahepatic or extrahepatic biliary system, is a rare but devastating malignancy. Prognosis is poor, with 5-year overall survival <5% including patients undergoing surgery. Resection is the only curative treatment; however, only ∼30% of patients present at a resectable stage, and intrahepatic recurrence is common even after complete resection. This article discusses the current role of transarterial therapies in the treatment of intrahepatic cholangiocarcinoma.
Surgical Aspects of Intrahepatic Cholangiocarcinoma
AbstractIntrahepatic cholangiocarcinoma (ICC) is a rare and aggressive malignancy. It originates from the bile ducts and is the second most common primary cancer of the liver. Surgery is considered the only curative treatment of ICC, offering the best chance for long-term survival. The purpose of this article is to review the available literature on ICC, with a focus on the various aspects of the surgical care in this potentially lethal malignancy.
New England Journal of Medicine · 2021 · 6 citations
Case 8-2021: A 34-Year-Old Woman with Cholangiocarcinoma
AbstractA 34-year-old woman was evaluated in the oncology clinic for the management of recurrent, metastatic intrahepatic cholangiocarcinoma. Before evaluation, an extended right hepatectomy had been performed, and the patient had received adjuvant therapy with gemcitabine and cisplatin. When relapse of the cancer occurred, additional management decisions were made.
DOAJ (DOAJ: Directory of Open Access Journals) · 2021 · 0 citations · open access
Advance in Therapeutic Targets of Intrahepatic Cholangiocarcinoma
AbstractIntrahepatic cholangiocarcinoma (ICC) is highly invasive and has poor prognosis. At present, there is no effective adjuvant treatment. With the indepth researches on ICC-related tumor microenvironment, gene, protein, epigenetic modification and signaling pathway, the potential therapeutic targets have been found. This article will review the novel potential therapeutic targets of ICC.
Progress of targeted therapy of intrahepatic cholangiocarcinoma
AbstractThe incidence of intrahepatic cholangiocarcinoma (ICC) has been increasing year by year. For most patients, surgical resection is not suitable when they are diagnosed as ICC. Conventional chemotherapy and radiotherapy are not effective for the long-term survival rate of ICC patients and lead to the poor overall prognosis. In recent years, with the deepening understanding about the molecular mechanism of biliary malignant tumors, some key genes and signaling pathways related to the pathogenesis of ICC have been identified, providing new ideas for the targeted therapy. In this paper, major molecular mechanisms and targeted therapies of ICC are reviewed.
Key words:
Bile duct neoplasm; Cholangiocarcinoma; Intrahepatic cholangiocarcinoma; Molecular targeted therapy; Progress
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
