DeCure for Autosomal dominant polycystic liver disease
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for autosomal dominant polycystic liver disease — screening already-approved drugs against its 19-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAutosomal dominant polycystic liver disease maps to a 19-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 autosomal dominant polycystic liver disease 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
RuvB like AAA ATPase 1 (RUVBL1) — RUVBL1 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 adpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2C9O · 2.2 Å · ligand ADENOSINE-5'-DIPHOSPHATE (ADP). Experimental structure, not a prediction.
What the evidence adds up to
The incidence rate of definite and likely isolated autosomal-dominant polycystic liver disease in Olmsted County, Minnesota, from 1980 to 2016 was 1.01 per 100,000 person-years, and the point prevalence on 1 January 2010 was 9.5 per 100,000 population. Only 15 of 35 definite and likely incident cases had received a diagnostic code, and only 8 had clinically significant hepatomegaly. When possible cases identified through radiology databases were added, incidence rates were much higher, particularly in recent years and in older patients, due to increased use of abdominal imaging. The authors conclude that clinically significant isolated ADPLD is rare, with a prevalence below 1 in 10,000, but that the overall prevalence, largely not clinically significant, is likely much higher and closer to the reported genetic prevalence of 1 in 496 for truncating mutations in ADPLD genes.
The clinical course of ADPLD is heterogeneous and dictated by the extent and severity of hepatic cysts and, when part of autosomal-dominant polycystic kidney disease, extrahepatic manifestations. Current management is directed toward palliating symptoms and treating complications. A 2025 case report describes a 53-year-old man incidentally found to have ADPLD after presenting with symptoms, and notes that treatment is limited, with surgical interventions and medical management being the mainstays. The report states that early diagnosis and management are essential to mitigate symptoms and improve patient outcome.
No drug treatment is mentioned in any of these abstracts. The 2010 review states that management is directed toward palliating symptoms, and the 2025 case report repeats that treatment is limited to surgery and medical management without specifying any drug. There is no evidence from these abstracts for any pharmacological intervention that alters the natural history of the disease.
What is still missing is any randomised controlled trial of a drug for isolated ADPLD, any validated patient stratification system to predict who will progress from asymptomatic to symptomatic disease, and dedicated funding for such trials, given the rarity of clinically significant cases.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
JHEP Reports · 2020 · 25 citations · open access
Epidemiology of autosomal-dominant polycystic liver disease in Olmsted county
AbstractBACKGROUND & AIMS: Isolated autosomal-dominant polycystic liver disease (ADPLD) is generally considered a rare disease. However, the frequency of truncating mutations to ADPLD genes in large, population sequencing databases is 1:496. With the increasing use of abdominal imaging, incidental detection of hepatic cysts and ADPLD has become more frequent. The present study was performed to ascertain the incidence and point prevalence of ADPLD in Olmsted County, MN, USA, and how these are impacted by the increasing utilisation of abdominal imaging. METHODS: The Rochester Epidemiology Project and radiology databases of Mayo Clinic and Olmsted Medical Center were searched to identify all subjects meeting diagnostic criteria for definite, likely, or possible ADPLD. Annual incidence rates were calculated using incident cases during 1980-2016 as numerator, and age- and sex-specific estimates of the population of Olmsted County as denominator. Point prevalence was calculated using prevalence cases as numerator, and age- and sex-specific estimates of the population of Olmsted County on 1 January 2010 as denominator. RESULTS: The incidence rate and point prevalence of combined definite and likely ADPLD were 1.01 per 100,000 person-years and 9.5 per 100,000 population, respectively. Only 15 of 35 definite and likely incident ADPLD cases had received a diagnostic code, and only 8 had clinically significant hepatomegaly. The incidence rates were much higher when adding possible cases, mainly identified through radiology databases, particularly in recent years and in older patients because of the increased utilisation of imaging studies. CONCLUSIONS: Clinically significant isolated ADPLD is a rare disease with a prevalence <1:10,000 population. The overall prevalence of ADPLD, however, to a large extent not clinically significant, is likely much higher and closer to the reported genetic prevalence. LAY SUMMARY: Isolated autosomal-dominant polycystic liver disease (ADPLD) is generally considered a rare disease. However, we demonstrate that it is a relatively common disease, which is rarely (<1:10,000 population) clinically significant.
AbstractAutosomal dominant polycystic liver disease (ADPLD) can occur as an independent disease entity affecting primarily the liver with few extrahepatic manifestations or as part of the disease spectrum of autosomal dominant polycystic kidney disease (ADPKD). The clinical course of ADPLD is heterogeneous and dictated by the extent and severity of hepatic and, in the case of ADPKD, extrahepatic manifestations. Currently, the management of ADPLD is directed toward palliating symptoms and treating complications.
Annals of Internal Medicine Clinical Cases · 2025 · 1 citations · open access
A Glimpse Into Isolated Polycystic Liver Disease: Image Case
AbstractAutosomal-dominant polycystic liver disease is a rare genetic disorder characterized by the formation of cysts on the liver that can progress and replace liver tissue. It leads to symptoms such as early satiety, compression of adjacent organs, and abdominal discomfort. While some patients may remain asymptomatic, others develop symptoms that significantly impact their quality of life. We present a 53-year-old man who presented with symptoms and was incidentally found to have autosomal-dominant polycystic liver disease. Treatment is limited, with surgical interventions and medical management being the mainstays. Early diagnosis and management are essential to mitigate symptoms and improve patient outcome.
Polycystic Liver Disease Presenting as Portal Hypertension
AbstractAutosomal dominant polycystic liver disease (PCLD) is a systemic hereditary disorder associated with cyst formation in the ductal organs like kidney and liver. Portal hypertension as a presenting manifestation is very rare. We report a case of 58-year-old male with PCLD presenting with portal hypertension and impaired hepatocellular function, managed conservatively.
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.
