DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for cystic kidney disease — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCystic kidney disease maps to a 34-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 cystic 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
Cystinosis is an autosomal recessive lysosomal storage disorder in which intracellular cystine accumulation leads to crystal formation in many tissues, including the placenta. A 1988 case report describes a successful pregnancy in a woman with nephropathic cystinosis who had a renal allograft; placental cystine crystals were noted, but the pregnancy outcome was not compromised. Before renal transplantation, female patients with cystinosis did not survive past adolescence, so reproductive capacity was previously unknown.
Cystine-depleting therapy with cysteamine and renal allograft transplantation have changed the natural history of cystinosis. Without treatment, renal function deteriorates to end-stage renal disease by the end of the first decade of life. The immediate-release formulation of cysteamine bitartrate (Cystagon) requires strict six-hourly dosing and has significant side effects; non-adherence is a major problem. A delayed-release enteric-coated formulation (Procysbi) was approved by the European Medicines Agency for twice-daily dosing, and an ongoing long-term clinical trial is testing its safety and efficacy. Longer survival now makes transition from paediatric to adult care a new challenge.
Neonatal renal cystic diseases, both hereditary and non-hereditary, cause severe morbidity and mortality. The main diagnostic tool is ultrasound, and most cases are detected on prenatal screening. Commonly encountered conditions include autosomal dominant polycystic kidney disease, autosomal recessive polycystic kidney disease, and multicystic dysplastic kidney. A 2005 review notes that renal cystic diseases are a heterogeneous group of heritable, developmental, and acquired disorders, and that definitive diagnosis requires clinical, radiological, pathological, and genetic analysis for accurate prognosis and genetic counselling.
What is still missing is a therapy that reverses or halts cyst formation in the common cystic kidney diseases; the cysteamine data apply only to cystinosis, a rare cause of renal cysts. No randomised trial has tested cysteamine in autosomal dominant or recessive polycystic kidney disease. The delayed-release cysteamine trial is ongoing, and its long-term effect on renal survival in cystinosis is not yet reported. For the more prevalent cystic kidney diseases, no disease-modifying drug has been proven in a controlled trial.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
New England Journal of Medicine · 1988 · 57 citations
Successful Pregnancy despite Placental Cystine Crystals in a Woman with Nephropathic Cystinosis
AbstractCYSTINOSIS is an autosomal recessive disorder characterized by intracellular accumulation of cystine due to failure of the normal carrier-mediated system that transports cystine out of lysosomes.1 , 2 Intracellular cystine storage results in crystal formation in many tissues of the body. Clinical findings include growth retardation, photophobia, renal tubular Fanconi's syndrome, and renal glomerular failure, generally occurring by the age of 10 years.3 , 4 Before the era of renal transplantation,5 female patients with cystinosis did not survive past adolescence, and their reproductive capacity was unknown. We report a successful pregnancy in a woman with cystinosis and a renal allograft, along with the unusual . . .
AbstractCystinosis is a rare, autosomal recessive inherited lysosomal storage disease. It is the most frequent and potentially treatable cause of the inherited renal Fanconi syndrome. If left untreated, renal function rapidly deteriorates towards end-stage renal disease by the end of the first decade of life. Due to its rarity and non-specific presentation, the entity is often not promptly recognized resulting in delayed diagnosis. Two major milestones in cystinosis management, cystine-depleting therapy with cysteamine and renal allograft transplantation, have had a considerable impact on the natural history and prognosis of cystinosis patients. However, due to its significant side effects and a strict 6-hourly dosing regimen, non-adherence to the immediate release of cysteamine bitartrate formulation (Cystagon®) is a major issue that might affect long-term outcome. Recently, a new twice-daily administered delayed-release enteric-coated formula of cysteamine bitartrate (Procysbi(TM)) has been approved by the European Medical Agency for the treatment of cystinosis, and has been shown to be safe and effective. This delayed-release cysteamine has the potential to improve compliance and hence prognosis, through its better dosing regimen, positive impact on quality of life and possibly less side-effects, and is now tested in an ongoing long-term clinical trial. Longer survival of patients with cystinosis makes transition from pediatric to adult-oriented care another challenge in cystinosis management and requires an extended multidisciplinary approach.
The Journal of Maternal-Fetal & Neonatal Medicine · 2017 · 16 citations
Neonatal renal cystic diseases
AbstractPURPOSE: Neonatal renal cystic diseases have a great impact on the morbidity and mortality of the affected neonates and infants. A good insight into the pathophysiology, diagnosis and treatment options of various neonatal renal cystic diseases aid in early diagnosis and intervention, thereby preventing complications. METHODS: PubMed search was done for articles on "neonatal renal cystic diseases" and relevant publications including reviews were considered for our article. RESULTS: Both hereditary and nonhereditary causes of cystic kidney diseases can result in severe morbidity and mortality. The main diagnostic modality is ultrasound imaging and most of the neonatal renal cystic diseases are detected during prenatal ultrasound screening. Commonly encountered neonatal renal cystic diseases are autosomal dominant polycystic kidney disease, autosomal recessive polycystic kidney disease and multicystic dysplastic kidney. CONCLUSIONS: A thorough knowledge of various renal cystic diseases can be of extreme prognostic value. Physicians should be aware of the impact of early diagnosis and intervention on the lives of those affected. Further research about treatment of these diseases is ongoing and can result in breakthrough therapies for these patients.
Quelle attitude lors de la découverte de kystes rénaux ?
AbstractEvaluation and management of renal cysts Renal cystic diseases are a heterogeneous group of conditions including heritable, developmental, and acquired disorders. They are united by the presence of microscopic or giant fluid-filled cavities and affect both children and adults. The definitive diagnosis of many of the renal cystic diseases requires clinical, radiological, pathological, and genetic analysis. A precise diagnosis is essential for prognosis, treatment, and future genetic counselling.
Journal of Medical Science And clinical Research · 2019 · 0 citations · open access
The Use of Bicarbonate Therapy in Critical Care for Metabolic Acidosis
AbstractMetabolic acidosis is a condition when there is a relative accumulation of plasma anions than the cations, which reduces the plasma pH. Replacement by sodium bicarbonate therapy is useful for the patients with diarrhea or renal tubular acidosis, but there is no definite evidence that sodium bicarbonate administration to the patients with acute metabolic acidosis. Acute metabolic acidosis occurs in conditions like diabetic ketoacidosis, lactic acidosis, septic shock, intraoperative metabolic acidosis. Patients with advanced chronic kidney disease also shows metabolic acidosis due to increased unmeasured anions and hyperchloremia. It's also a predominant buffer used in dialysis fluids anda load of sodium bicarbonate subjected to patients on maintenance dialysis during the sessions, suffering a transient metabolic alkalosis of variable severity. Side effects associated with sodium bicarbonate therapy include hypokalemia, ionized hypocalcemia, hypercapnia, and QTc interval prolongation. Administration of sodium bicarbonate in sepsis that is subject to ongoing debate.
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.
