DeCure for Polycystic liver disease 4 with or without kidney cysts
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for polycystic liver disease 4 with or without kidney cysts — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePolycystic liver disease 4 with or without kidney cysts maps to a 2-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 polycystic liver disease 4 with or without kidney cysts 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
polycystin 1, transient receptor potential channel interacting (PKD1) — PKD1 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 1rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8ZKH · 2.3 Å · ligand (1R)-2-{[(S)-{[(2S)-2,3-dihydroxypropyl]oxy}(hydroxy)phosphoryl]oxy}-1-[(hexadecanoyloxy)methyl]ethyl
(9Z)-octadec-9-enoate (PGW). Experimental structure, not a prediction.
What the evidence adds up to
Polycystic liver disease (PLD) is defined by the presence of more than ten cysts in the liver. It is a rare genetic condition that can occur as an isolated disease or together with polycystic kidney disease. In one autopsy series from UCLA Medical Center, the prevalence of adult polycystic liver disease was 0.13%; 93% of those cases also had polycystic kidney disease, and 45% of patients with adult polycystic kidney disease had associated liver cysts. The pathogenesis involves ductal plate malformation, ciliary dysfunction, and changes in cell signalling. Most patients are asymptomatic, but in 2–5% of cases the disease causes disabling symptoms and a significant reduction in quality of life.
A 1988 case report describes a patient with unusually severe hepatomegaly — a liver weighing 7.7 kg with a volume of 22,080 cm³ — associated with Von Meyenburg's complexes, which are thought to be aberrant embryonic intrahepatic bile ducts. Progression to cirrhosis is rare; a 2021 case report notes that data on this complication are sparse and that the only curative treatment is liver transplantation.
Somatostatin analogues are described as holding promise for controlling disease progression, but the same 2022 review states that liver transplantation remains the only curative treatment modality. No randomised controlled trials, response rates, or survival data from drug interventions are reported in these abstracts.
What is still missing are large, prospective trials of somatostatin analogues or other medical therapies with clearly defined endpoints, adequate funding for such trials, and better patient stratification to identify the small subset of symptomatic patients who might benefit before transplantation becomes necessary.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The American Journal of Surgical Pathology · 1988 · 87 citations
Massive Hepatomegaly in Adult Polycystic Liver Disease
AbstractIn adult polycystic liver disease, the liver gradually enlarges as it is replaced by cysts. The disease rarely produces symptoms or complications. Liver cysts are thought to arise from aberrant embryonic intrahepatic bile ducts (Von Meyenburg's complexes). We present a case of adult polycystic liver disease with Von Meyenburg's complexes and unusually severe hepatomegaly (7.7 kg, 22,080 cm3). The autopsy prevalence of adult polycystic liver disease at UCLA Medical Center is 0.13%; 93% of these cases had polycystic kidney disease. Adult polycystic kidney disease had associated liver cysts in 45% of cases.
Hepatic Medicine Evidence and Research · 2022 · 27 citations · open access
Polycystic Liver Disease: Pathophysiology, Diagnosis and Treatment
AbstractPolycystic liver disease (PLD) is a clinical condition characterized by the presence of more than 10 cysts in the liver. It is a rare disease Of genetic etiology that presents as an isolated disease or assoc\iated with polycystic kidney disease. Ductal plate malformation, ciliary dysfunction, and changes in cell signaling are the main factors involved in its pathogenesis. Most patients with PLD are asymptomatic, but in 2-5% of cases the disease has disabling symptoms and a significant reduction in quality of life. The diagnosis is based on family history of hepatic and/or renal polycystic disease, clinical manifestations, patient age, and polycystic liver phenotype shown on imaging examinations. PLD treatment has evolved considerably in the last decades. Somatostatin analogues hold promise in controlling disease progression, but liver transplantation remains a unique curative treatment modality.
Canadian Journal of Gastroenterology · 2004 · 9 citations · open access
Management of Polycystic Liver Disease
AbstractPolycystic liver disease (PCLD) is characterized by multiple cysts throughout the liver. Patients may develop chronic intractable symptoms that may be debilitating. Others may develop medical complications that necessitate intervention. There is a variety of nonsurgical and surgical treatment options for symptomatic or complicated PCLD, which range from cyst aspiration and fenestration to liver transplantation. Studies have described variable efficacy and morbidity. Currently, there are no guidelines for the management of PCLD patients and the optimal intervention is controversial. This article reviews the pathogenesis, classification and spectrum of treatment options for PCLD.
Tropical Journal of Medical Research · 2017 · 1 citations · open access
Massive polycystic liver disease in autosomal-dominant polycystic kidney disease
AbstractPolycystic liver diseases (PLDs) are a rare group of genetic disorders, in which multiple cysts occur in the liver. This could occur on a background of cystic kidneys, known as autosomal-dominant polycystic kidney disease (ADPKD) or without the presence of cysts in any other organ of the body, known as autosomal-dominant PLD. Irrespective of the type of the polycystic liver, the natural course of these disorders is similar. Both genders are affected, but the female gender has a higher prevalence. Majority of patients with PLD are asymptomatic and can be managed conservatively, but a minority could develop massive hepatomegaly and attendant symptoms from compression of the surrounding organs. We present a patient with massive polycystic liver on a background of ADPKD.
Greater South Information System · 2021 · 0 citations · open access
Hepatorenal Polycystosis Complicated By Hepatic Cirrhosis: A Case Report
AbstractPolycystic liver disease is most commonly associated with autosomal dominant polycystic kidney disease. Hepatic cysts are the most common extrarenal manifestation of autosomal dominant polycystic kidney disease. The progression to cirrhosis remains rare, and the data is sparse, the only curative treatment is liver transplantation. We report the case of a young patient with hepato-renal polycystosis at the stage of cirrhosis.
International Journal of Innovative Research in Medical Science · 2021 · 0 citations · open access
Hepatorenal Polycystosis Complicated By Hepatic Cirrhosis: A Case Report
AbstractPolycystic liver disease is most commonly associated with autosomal dominant polycystic kidney disease. Hepatic cysts are the most common extrarenal manifestation of autosomal dominant polycystic kidney disease. The progression to cirrhosis remains rare, and the data is sparse, the only curative treatment is liver transplantation. We report the case of a young patient with hepato-renal polycystosis at the stage of cirrhosis.
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.
