DeCure for Polycystic liver disease 3 with or without kidney cysts
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for polycystic liver disease 3 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 3 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 3 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 10 cysts in the liver and is a rare genetic condition that can occur alone or together with polycystic kidney disease. In a 1988 study of 66 consecutive patients with adult polycystic kidney disease who all underwent CT scanning, a clear correlation was found between age and the frequency of hepatic cysts. The study also found that liver cysts continue to grow and appear de novo even after renal replacement therapy begins, unlike kidney cysts. Most patients with PLD are asymptomatic, but in 2–5% of cases the disease causes disabling symptoms and a significant reduction in quality of life.
Somatostatin analogues are described as holding promise for controlling disease progression, but liver transplantation remains the only curative treatment. A 2023 case report describes a 57-year-old man with polycystic kidneys diagnosed at age 24 and liver cysts discovered at age 38. His CT scan showed a 33 × 21 × 27 cm polycystic liver with cysts up to 7 cm in diameter. Between 2009 and 2019 he underwent repeated cyst punctures, but because the liver continued to enlarge and his complaints worsened, he was placed on a transplant waiting list in spring 2019. He received a liver transplant on 11 July 2022; the explanted liver measured 53 × 37 × 39 × 16 cm and weighed 14.75 kg. The postoperative course was uneventful, and the authors state that liver transplantation can be a very effective treatment that significantly improves quality of life.
Progression to cirrhosis in PLD is rare, and data on it are sparse. A 2021 case report describes a young patient with hepato-renal polycystosis who reached the stage of cirrhosis, and again notes that the only curative treatment is liver transplantation. No drug other than somatostatin analogues is mentioned in these abstracts, and no quantitative efficacy data (response rates, survival figures) are provided for any pharmacological intervention. What remains missing are prospective trials that test somatostatin analogues against placebo in stratified patient groups, long-term outcome data beyond single case reports, and dedicated funding for such studies in this rare disease.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Acta Medica Scandinavica · 1988 · 56 citations
Frequency of Hepatic Cysts in Adult Polycystic Kidney Disease
AbstractThe frequency of hepatic cysts in patients with adult polycystic kidney disease (APKD) is usually considered to average about 33%, but with great, and hitherto largely unexplained, variations between different studies. Sixty-six consecutive patients with APKD were all subjected to CT scanning of the liver and of the kidneys. In confirmation of one previous study, a clearcut correlation was found between age (= duration of disease) and the frequency of hepatic cysts. It was furthermore found that cysts in the liver, as opposed to cysts in the kidneys, continue to grow and appear de novo even after institution of renal replacement therapy.
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.
Journal of Surgical Case Reports · 2023 · 1 citations · open access
Liver transplantation for polycystic liver disease: a case report
AbstractA 57-year-old man complained about abdominal distension and pain, constant feeling of early satiety. He was diagnosed with polycystic kidneys at the age of 24 and liver cysts discovered at the age of 38. The CT scan revealed 33 x 21 x 27 cm polycystic liver with cysts up to 7 cm in diameter. In 2009-2019 the patient was repeatedly punctured for liver cysts. Considering the continued enlargement of the liver and the worsening of complaints, the patient was put on the waiting list for a liver transplant in the spring of 2019. The patient went through liver transplantation on 11th of July 2022, the liver measures were 53 x 37 x 39 x 16 cm and weight 14,75 kg. The postoperative course was uneventful. Liver transplantation can be very effective treatment method that significantly improves the quality of life in PLD patients.
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.
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.
Oxford University Press eBooks · 2015 · 0 citations
Management of cystic liver disease
AbstractAbstract In a subset of autosomal dominant polycystic kidney disease patients, hepatic cysts dominate the clinical picture. These patients may develop polycystic liver disease, and enlargement of the liver leads to compression of adjacent abdominal and thoracic organs. The main risk factors for growth of liver cysts are female sex, exogenous oestrogen use, multiple pregnancies, and severity of renal disease. Treatment is only indicated in those with symptoms, and choice of treatment depends on total liver volume, size, and location of the liver cysts. Current radiological and surgical therapies include aspiration-sclerotherapy, fenestration, segmental hepatic resection, and liver transplantation. They all are palliative in nature and are partially effective and have non-negligible morbidity and mortality. Somatostatin analogues are still in development for polycystic liver disease.
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.
