DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for pediatric hepatocellular carcinoma — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePediatric hepatocellular carcinoma maps to a 1-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
approvedSunitinibApproved drug
Structures already discussed alongside pediatric hepatocellular carcinoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
KIT kinase domain — Sunitinib has a real, experimentally solved structure in complex with this target (PDB 3G0E, 1.6 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 b49drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3G0E · 1.6 Å · ligand Sunitinib (B49). Experimental structure, not a prediction.
What the evidence adds up to
Pediatric hepatocellular carcinoma is a rare malignant liver tumour in children and adolescents, usually advanced at presentation and poorly responsive to chemotherapy. Overall survival figures for children with HCC are 20–40%, and for unresectable tumours the five-year survival rate is less than 20%. Only 20–30% of children can achieve complete surgical resection, which is frequently limited by tumour size, location, extent, and underlying liver disease. The tumour shows low sensitivity to standard chemotherapy, and innovative approaches including targeted and immunotherapy are being considered but remain investigational.
Liver transplantation is indicated for unresectable HCC localised to the liver. After total hepatectomy and transplantation, patient survival is 70–80% at one year and 60–70% at five years. Recurrent or metastatic tumour accounts for the majority of mortality after transplant. Results remain poor when HCC is advanced with major vascular invasion or extrahepatic spread at presentation. An exception is children whose HCC is detected during surveillance of chronic liver disease; they typically have smaller tumours and a good prognosis after transplantation. The role of transplantation in other primary hepatic malignancies is uncertain because experience is very limited.
A 2019 case report describes a 55-year-old man with primary clear cell carcinoma of the liver (a rare subtype of HCC) who developed multiple metastases after radical excision. He was treated with sunitinib 37.5 mg orally once daily combined with Chinese herbal medicine. Tumour size steadily reduced, lesions were no longer obvious after about two years, and the patient was considered clinically cured, with survival of eight years from initial diagnosis. This is a single adult case, not a paediatric trial, and no conclusions can be drawn for children.
What is still missing are prospective paediatric trials that define which tumour and patient characteristics predict benefit from transplantation, and whether targeted or immunotherapy agents have any meaningful activity in paediatric HCC. The rarity of the disease makes trial recruitment difficult, and no standard systemic therapy for unresectable paediatric HCC has been established. Patient stratification by molecular subtype, underlying liver disease, and extent of vascular invasion is needed before any treatment can be recommended.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Annals of The Royal College of Surgeons of England · 2007 · 56 citations · open access
The Role of Liver Transplantation in the Management of Paediatric Liver Tumours
AbstractIn recent years, considerable progress has been made in the treatment of children with hepatoblastoma largely due to effective pre-operative chemotherapy. Total hepatectomy and liver transplantation has emerged as an effective treatment for the small proportion of children with unresectable hepatoblastoma limited to the liver. A 5-year survival of 70% can be achieved in such cases. In contrast, the results of liver transplantation in children with hepatocellular cancer remain poor because these tumours are usually advanced with evidence of major vascular invasion and/or extrahepatic spread at the time of presentation. An exception is those children in whom the hepatocellular carcinoma is detected during surveillance of chronic liver disease - they typically have smaller tumours and frequently have a good prognosis after liver transplantation. The role of liver transplantation in children with other primary hepatic malignancies remains uncertain because experience is very limited. Liver transplantation is rarely needed in the management of children with benign liver tumours but, if other treatments have failed, it can be a life-saving intervention.
International Journal of Molecular Sciences · 2025 · 9 citations · open access
Pediatric Hepatocellular Carcinoma: A Review of Predisposing Conditions, Molecular Mechanisms, and Clinical Considerations
AbstractPediatric hepatocellular carcinoma (HCC) is a rare malignant liver tumor affecting children and adolescents and occurring either sporadically or in the context of underlying liver disease. In this review, we detail the epidemiology of pediatric HCC with a focus on predisposing factors including hepatic or systemic disease, genetic disorders, and familial cancer syndromes. We summarize existing research on the pathophysiology of pediatric HCC, including molecular mechanisms of oncogenesis, highlighting unique disease features differentiating pediatric HCC from adult HCC. We then survey the landscape of therapeutic options for pediatric HCC, including novel therapeutics. Lastly, we discuss the pathologic spectrum upon which pediatric HCC is postulated to exist, ranging from hepatoblastoma to HCC and including the hybrid entity hepatocellular neoplasm not otherwise specifed (HCN-NOS). In summary, we highlight the key clinical and molecular features of pediatric HCC that may inform future research and novel approaches to the clinical care of these patients.
OncoTargets and Therapy · 2019 · 3 citations · open access
<p>Sunitinib-and-Chinese herbal medicine-based systematic treatment clinically cured a patient with multiple metastatic primary clear cell carcinoma of the liver: a case report</p>
AbstractPrimary clear cell carcinoma of the liver (PCCCL) is a rare and special type of primary hepatocellular carcinoma. However, treatment methods for multiple metastatic PCCCL are lacking. Here, we report the case of a 55-year-old male PCCCL patient with multiple metastatic lesions who was clinically cured by sunitinib-based systematic treatment. This patient was diagnosed with PCCCL in Liver Segment 7, Child-Pugh A liver function, Stage A in November 16, 2009, and received radical excision of the cancer immediately. His disease recurred with multiple metastatic lesions in the liver and other parts of the body, including the retroperitoneal lymph nodes, lung and bilateral adrenal nodules in June 29, 2012. The biopsy results showed that the lung mass was lung metastasis of PCCCL. With Child-Pugh A liver function, Stage C of PCCCL was diagnosed. Sunitinib (37.5 mg, oral, once a day [qd]) in combination with Chinese herbal medicine (CHM) was given. The tumor size steadily reduced, and the lesions were no longer obvious in May 21, 2014. The patient had multiple metastases and is in complete response (CR) state until now. He is considered as clinically cured. From the initial diagnosis of PCCCL, the survival period reached 8 years.
Current Opinion in Organ Transplantation · 2006 · 0 citations
Liver transplantation for hepatocellular carcinoma in children
AbstractPurpose of review Hepatocellular carcinoma is a relatively uncommon tumor in children which is usually advanced in stage at presentation and responds poorly to chemotherapy. Optimal treatment includes complete surgical resection. The role of liver transplantation in the treatment of unresectable hepatocellular carcinoma in children is not fully defined. The purpose of this review is to discuss the important and unique aspects of hepatocellular carcinoma in children and review the available outcome data on liver transplantation for hepatocellular carcinoma in children. Recent findings Overall survival of children with hepatocellular carcinoma is 20–40%. Complete surgical resection is frequently limited by tumor size, location, extent, and underlying liver disease. Only 20–30% of children may actually be able to achieve complete resection. Total hepatectomy and liver transplantation are indicated for unresectable hepatocellular carcinoma localized to the liver, with patient survival of 70–80% at 1 year and 60–70% at 5 years. Recurrent or metastatic tumor accounts for the majority of patient mortality after transplant. Summary Current outcomes of liver transplantation for unresectable hepatocellular carcinoma in children are encouraging. Further studies are needed to define tumor and patient characteristics that will optimize the role of liver transplantation in pediatric hepatocellular carcinoma.
Pediatric Hematology/Oncology and Immunopathology · 2025 · 0 citations · open access
Hepatocellular carcinoma in children and adolescents: current status
AbstractHepatocellular carcinoma (HCC) is a very rare primary liver tumor in children and adolescents associated with an aggressive clinical course and unfavorable prognosis. The five-year overall survival rate for unresectable tumors is less than 20%, which underscores the importance of early diagnosis and radical surgery. This review is dedicated to current issues and challenges in the treatment of pediatric HCC. Here, we provided epidemiological data including information on risk factors for HCC and discussed molecular and genetic features of the tumor, its diagnosis and extent assessment. We also included HCC treatment outcomes from prospective clinical trials as well as current standards of therapy in children and adolescents. Finally, special attention was paid to an analysis of results of various surgical treatments including resections and liver transplantation. We reported low tumor sensitivity to standard chemotherapy and discussed innovative approaches to disease management in patients with unresectable tumors as well as contemplated the prospect of using targeted and immunotherapy in pediatric patients. The review also highlights the importance of a multidisciplinary approach, timely diagnosis, and the introduction of innovative treatments for the improvement of therapy outcomes.
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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