DeCure for Autosomal recessive polycystic kidney disease
DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for autosomal recessive polycystic kidney disease — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAutosomal recessive polycystic kidney disease maps to a 3-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 recessive polycystic kidney 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
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
The three abstracts provided all concern autosomal dominant polycystic kidney disease (ADPKD), not the recessive form. No abstract addresses autosomal recessive polycystic kidney disease (ARPKD). The 2022 review states that ADPKD accounts for 10% of end-stage kidney disease patients and has an estimated global prevalence of 12.5 million cases. It notes that tolvaptan has received regulatory approval for ADPKD patients at risk of rapid progression to kidney failure. The 1998 abstract describes ADPKD as a prevalent human genetic disorder and reports progress in understanding its genetic basis and cellular mechanisms. The 2019 abstract from Senegal reports that ADPKD is poorly reported in black patients and that some data suggest the disease is exceptional in this group; it aimed to investigate epidemiological, clinical, and evolutionary characteristics of ADPKD cases in three semi-urban centres. The 1992 abstract notes that ADPKD is increasingly diagnosed in childhood due to improved ultrasonography, and that early treatment of hypertension may slow disease progression; it also states that autosomal recessive polycystic kidney disease is the more commonly symptomatic disease in childhood.
No abstract provides any data on drug repurposing, response rates, survival, or sample sizes for any treatment in ARPKD. No abstract mentions any drug other than tolvaptan, which is approved for ADPKD, not ARPKD. The 1992 abstract expresses anticipation of innovative therapeutic strategies and improved survival, but offers no concrete results.
What is still missing for ARPKD: any clinical trial data, any drug repurposing evidence, any patient stratification strategy, and any dedicated funding for research into this specific disease. The available literature focuses almost entirely on the dominant form.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Kidney Research and Clinical Practice · 2022 · 31 citations · open access
Management of autosomal dominant polycystic kidney disease in the era of disease-modifying treatment options
AbstractAutosomal dominant polycystic kidney disease (ADPKD) is the reported etiology in 10% of end-stage kidney disease (ESKD) patients and has an estimated prevalence of 12.5 million cases worldwide across all ethnicities. There have been major advancements over the last two decades in understanding the pathogenesis and development of disease-modifying treatment options for ADPKD, culminating in regulatory approval of tolvaptan for ADPKD patients at risk of rapid progression to kidney failure. This review highlights the genetic mutations associated with ADPKD, defines patients at risk of rapid progression to ESKD, and focuses on the management of ADPKD in the era of disease-modifying agents.
Fetal and Pediatric Pathology · 1998 · 3 citations
Molecular Biology of Autosomal Dominant Polycystic Kidney Disease
AbstractAmong the prevalent human genetic disorders, human autosomal dominant polycystic kidney disease is certainly one of the most challenging, both from a clinical and a fundamental perspective. In the recent years, important progress opened novel research avenues to elucidate the genetic basis, the cellular pathophysiologic mechanisms and the molecular function of genes and proteins involved in autosomal dominant polycystic kidney disease.
Kidney International Reports · 2019 · 0 citations · open access
SUN-264 AUTOSOMIC DOMINANT POLYCYSTIC KIDNEY DISEASE (ADPKD) IN BLACK AFRICAN PATIENTS : AN EXPERIMENT FROM THREE SEMI-URBAN CENTERS IN SENEGAL (WEST AFRICA)
AbstractAutosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary nephropathy in the world. ADPKD is poorly reported in the black patient and some data show that the disease is exceptional in the latter. Our purpose in this study was to investigate the epidemiological, clinical, para-clinical, therapeutic and evolutionary characteristics of ADPKD cases.
Current Opinion in Pediatrics · 1992 · 0 citations
Polycystic kidney diseases in childhood
AbstractAutosomal dominant polycystic kidney disease is increasingly diagnosed during childhood, primarily because of increased availability and sensitivity of ultrasonography, and less commonly because of symptoms. If early treatment of symptoms and signs, especially hypertension, can lead to diminution in the progression of the disease, it behooves pediatricians to be aware of the ramifications of this disease. Autosomal recessive polycystic kidney disease is the more commonly symptomatic disease in childhood. Information leading to the genetic loci and the pathogenetic features of these diseases is explored in this review, and clinical features and prenatal diagnostic considerations in autosomal dominant disease are discussed. Because of enhanced interest in these genetic renal diseases that lead to end-stage renal disease, we can anticipate innovative therapeutic strategies and improved survival in the future.
Effect of processing on the structure and properties of oxide dispersion strenghthened (ODS) γ1 hardened superalloys
AbstractAutosomal dominant polycystic kidney disease is the most common inherited kidney disease and accounts for 7–10% of all patients on renal replacement therapy worldwide. Although first reported 500 years ago, this disorder is still regarded as untreatable and its pathogenesis is poorly understood despite much study. During the past 40 years, however, remarkable advances have transformed our understanding of how the disease develops and have led to rapid changes in diagnosis, prognosis, and treatment, especially during the past decade. This Review will summarise the key findings, highlight recent developments, and look ahead to the changes in clinical practice that will likely arise from the adoption of a new management framework for this major kidney 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.
