DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for cholangiocarcinoma — screening already-approved drugs against its 47-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCholangiocarcinoma maps to a 47-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
approvedGemcitabineApproved drugapprovedRegorafenibApproved drug
Structures already discussed alongside cholangiocarcinoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
C4S dCK variant of dCK — Gemcitabine has a real, experimentally solved structure in complex with this target (PDB 2NO0, 1.8 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet geodrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2NO0 · 1.8 Å · ligand Gemcitabine (GEO). Experimental structure, not a prediction.
What the evidence adds up to
Cholangiocarcinoma is diagnosed late in most patients, and complete surgical resection is possible only in a minority of cases. For the majority, palliation is the mainstay. As of 2012, the recommended first-line regimen for advanced disease is gemcitabine combined with a platinum agent. A 2006 review described photodynamic therapy as "extremely promising" for adjunct treatment of intrahepatic mass lesions, and noted that long-term results from transplantation protocols for non-resectable localised disease had been reported. The same review identified hepatitis B and C, cirrhosis and alcohol as risk factors, and proposed a morphology-based classification to aid future studies.
A single case report from 2020 described prolonged survival with regorafenib in one patient with metastatic cholangiocarcinoma. The report explicitly states that advanced cholangiocarcinoma is a rare, aggressive tumour with limited treatment options and very few systemic therapies available. It notes that immune checkpoint inhibitors and tyrosine kinase inhibitors are being used as new approaches after first- or second-line chemotherapy, but provides no response rates or survival data from controlled trials.
The 2012 British Society of Gastroenterology update is based on randomised controlled trials, systematic reviews, meta-analyses, and cohort studies, but the abstract gives no numerical results for any intervention. A 2012 review of advanced disease states that treatments "are not very effective" except when complete surgical resection is possible, and that locoregional therapy is an interesting approach because cholangiocarcinomas have only a limited tendency to metastasise.
What is still missing are large, randomised trials that demonstrate a survival benefit for any systemic therapy beyond first-line gemcitabine-platinum. No drug has shown consistent, reproducible efficacy in a phase III setting for advanced disease. The field lacks validated biomarkers to stratify patients for targeted or immune-based treatments, and funding for adequately powered trials in this rare cancer remains insufficient.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Gut · 2012 · 794 citations · open access
Guidelines for the diagnosis and treatment of cholangiocarcinoma: an update
AbstractThe British Society of Gastroenterology guidelines on the management of cholangiocarcinoma were originally published in 2002. This is the first update since then and is based on a comprehensive review of the recent literature, including data from randomised controlled trials, systematic reviews, meta-analyses, cohort, prospective and retrospective studies.
Current Opinion in Gastroenterology · 2006 · 79 citations
Advances in the diagnosis, evaluation and management of cholangiocarcinoma
AbstractPURPOSE OF REVIEW: Cholangiocarcinoma is an enigmatic malignancy of the biliary tract that has recently been shown to be increasing in incidence globally. There has been recent progress in identifying potential risk factors for the tumor, and in the use of emerging technologies for diagnosis and palliative treatment. RECENT FINDINGS: Hepatitis B and C, cirrhosis and alcohol have been described as risk factors for cholangiocarcinoma. A morphology-based classification for cholangiocarcinoma is proposed that will facilitate future descriptive epidemiology, diagnosis and comparative therapeutic studies. Diagnosis may be improved by new approaches to enhance the diagnostic yield and utility of biliary cytology. The role of new imaging approaches such as positron emission tomography scanning, endoscopic ultrasound or optical coherence tomography for diagnosis are being examined and defined. Long-term results for transplantation protocols for curative intent in non-resectable localized disease have been described. Photodynamic therapy looks extremely promising for adjunct therapy of intrahepatic mass lesions. SUMMARY: Recent advances in the epidemiology, classification, diagnosis and therapy of cholangiocarcinoma are expected to enhance the evaluation and management of patients with this devastating malignancy.
World Journal of Gastroenterology · 2009 · 6 citations · open access
Repetitive response to gemcitabine that led to curative resection in cholangiocarcinoma
AbstractThis study reports a case of unresectable intrahepatic mass-forming cholangiocarcinoma which showed a dramatic response to gemcitabine that led to curative resection and a long-term survival of more than five years. Six and five cycles of gemcitabine monotherapy were administered separately over a three-year period and a radical excision was performed at 4.5 years after diagnosis. This case indicates the role of gemcitabine as a neoadjuvant chemotherapeutic agent for cholangiocarcinoma and guarantees a randomized controlled prospective study.
Diagnostic and Interventional Radiology · 2024 · 3 citations · open access
Hepatic arterial infusion chemotherapy combined with toripalimab and surufatinib for the treatment of advanced intrahepatic cholangiocarcinoma
AbstractPURPOSE: The aim of the present study is to report the clinical results of patients with advanced intrahepatic cholangiocarcinoma (ICC) who received combination therapy of hepatic arterial infusion chemotherapy (HAIC), toripalimab and surufatinib. METHODS: The study cohort consisted of 28 patients with advanced ICC who were treated with HAIC (mFOLFOX6 regimen, Q3W) in combination with intravenous toripalimab (240 mg, Q3W) and oral surufatinib (150 mg, once daily). The cohort had 14 male and 14 female patients. The baseline characteristics of the study cohort were obtained. The tumor response and drug-associated toxicity were assessed and reported. RESULTS: During the follow-up period (median follow-up time: 11.3 months; range: 4-19 months), four patients died of tumor progression. The objective response rate and disease control rate were 58% and 79%, respectively. The mPFS was 9.5 months, and the overall survival rate was 83.3%. The most frequent adverse events were nausea and vomiting (100%) and abdominal pain (85.7%). Serious complications related to death were not observed. CONCLUSION: The combination treatment schedule for advanced ICC demonstrated positive efficacy and safety profiles. CLINICAL SIGNIFICANCE: This study provides promising clinical guidance for the treatment of advanced cholangiocarcinoma and is expected to modify the treatment strategy for this disease.
EMJ Gastroenterology · 2012 · 2 citations · open access
Treatment of Advanced Cholangiocarcinoma: Current Status and Future Perspectives
AbstractCholangiocarcinoma (CC) is a tumour with a poor prognosis. The treatment of CC is challenging as the tumour is usually diagnosed late and the treatments are not very effective except when complete surgical resection is possible. However in the majority of cases, surgical resection is not possible and palliation is the mainstay of treatment. Currently chemotherapy combining gemcitabine with a platinum agent is the recommended first- line treatment regimen for advanced CC. Since, CCs have only a limited tendency to metastasize locoregional therapy is an interesting approach. The aim of this short review is to give an update on standard and prospective treatment concepts in advanced cholangiocarcinoma based on a comprehensive review of the recent literature and guidelines.
Advancements in Case Studies · 2020 · 0 citations · open access
Prolonged Survival with Regorafenib in Metastatic Cholangiocarcinoma: Case Report
AbstractAdvanced cholangiocarcinoma is a rare, aggressive tumor. Treatment is limited and very few systemic therapies are available. An effort for new drugs development is in course, which aims higher response rates, prolonged survival and better quality of life. Currently, immune checkpoint inhibitors and tyrosine kinase inhibitors have been used as new approaches after first- or second-line chemotherapy.
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.
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