DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome, type 20 — 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 moduleNephrotic syndrome, type 20 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 nephrotic syndrome, type 20 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
MORC family CW-type zinc finger 4 (MORC4) — MORC4 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 anpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7K7T · 2.94 Å · ligand PHOSPHOAMINOPHOSPHONIC ACID-ADENYLATE ESTER (ANP). Experimental structure, not a prediction.
What the evidence adds up to
Fifty-eight previously untreated adults with minimal-change nephrotic syndrome were given alternate-day steroids and followed for a mean of 35.8 months. Fifty-four patients (93%) achieved remission by 12 weeks and remained in remission at 16 weeks. Of those 54, eight (14.8%) had frequent relapses and nine (16.6%) had infrequent relapses. No serious complications from the steroid therapy were reported.
In children, nephrotic syndrome is predominantly minimal change disease. Untreated, it can cause infections, hypovolemia, hypercoagulability, hyperlipidemia, anaemia, and growth delays. Morbidity and occasional mortality arise from inadequate management and complications, not from renal failure itself.
A multicentre open-label trial randomised 172 children aged 1–4 years with a first episode of idiopathic nephrotic syndrome to either 3 months or 6 months of initial prednisone therapy. After a 6-week daily and 6-week alternate-day course, patients were randomised to either tapering prednisone on alternate days for 12 weeks or no further therapy. Over two years of follow-up, the proportions of patients in sustained remission and those with frequent relapses at one and two years were similar between groups. The hazard ratio for time to relapse was 0.75 (95% CI 0.53–1.06) and for frequent relapses was 0.78 (95% CI 0.52–1.18). Relapse rates and adverse event rates were also similar. The authors concluded that prolonged initial prednisolone therapy does not significantly alter the disease course in young children.
What remains missing is a trial design that can identify which, if any, subgroup of young children might benefit from longer initial steroid exposure, and funding to test alternative agents for the substantial fraction who relapse despite standard treatment.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Minimal-Change Nephrotic Syndrome in Adults Treated with Alternate-Day Steroids
AbstractFifty-eight previously untreated adults with minimal-change nephrotic syndrome (MCNS), who had a mean follow-up period of 35.8 months, were studied with regard to their response to alternate-day steroid therapy. The nephrotic syndrome in 54 patients (93%) remitted by 12 weeks and patients continued to be in remission at 16 weeks. Of the 54 patients, 8 (14.8%) had frequent relapses and 9 (16.6%) had infrequent relapses. No serious complications as a result of steroid therapy were encountered.
High-sulfated derivative of polysaccharide from <i>Ulva pertusa</i> improves Adriamycin-induced nephrotic syndrome by suppressing oxidative stress
Abstract). After treatment for 6 weeks, we assessed urine protein, renal function, and blood lipids, and observed morphology and histologic injury of the kidney and glomerular microstructure. Furthermore, we detected antioxidant enzyme activity and expression level of the Keap1/Nrf2 signaling pathway to explore the potential mechanism of HU. Results showed that HU not only alleviated hyperlipidemia and hypoalbuminemia, but also reduced urine protein by inhibiting podocyte detachment, thickening of the glomerular basement membrane, and expression of kidney fibrosis markers (collagens I and IV). In addition, HU enhanced antioxidant enzyme activity (GSH-Px, CAT, SOD) in both serum and the kidney, which may be due to upregulating the expression of Nrf2 and downregulating the expression of Keap1. In conclusion, HU appears to be effective in attenuating NS in rats through suppressing oxidative stress by regulating the Keap1/Nrf2 signaling pathway.
AbstractNephrotic syndrome is an important chronic disease in children, characterized by minimal change disease in the majority. Untreated nephrotic syndrome may cause numerous complications, such as, infections, hypovolemia, hypercoagulability, hyperlipidemia, anemia, growth and development delays. The morbidity and occasional mortality is from inadequate management and complications and not due to renal failure. Expertise and clinical judgement as well as cooperation of the family are crucial for optimal management and a favorable outcome.
Indian Journal of Research in Homoeopathy · 2022 · 2 citations · open access
Nephrotic syndrome treated with homoeopathy: An evidence-based case-report
AbstractIntroduction: Nephrotic syndrome (NS) is a glomerular disorder characterised by peripheral oedema, heavy proteinuria and hypoalbuminemia, often hyperlipidaemia. Patients generally present with oedema and fatigue. Complementary and alternative therapies have been widely used in the treatment of NS. The homoeopathic treatment for NS could be a safer approach to treating the disease. Case Summary: This is the case of a 32-year-old man who presented with NS with membranous nephropathy and was successfully managed with individualised homoeopathic medicine, Natrium Sulphuricum over 1 year. The case was followed up with documentation of clinical symptoms and investigation reports findings. Changes in the health status of the patient were measured by the ‘outcome in relation to impact on daily living instrument’ (ORIDL).
Journal of the American Society of Nephrology · 2021 · 1 citations
Randomized Controlled Trial Comparing 3- vs. 6-Months Initial Prednisone Therapy in Young (<4-Year-Old) Children with Nephrotic Syndrome
AbstractBackground: While recent RCTs suggest no role for prolonged (>2-3 months) initial corticosteroid therapy in NS, subgroup analysis in 2-studies suggests its association with reduced frequency of subsequent relapses in children <4-6 yr-old. This multicenter open-label trial examines the efficacy & safety of 3-months versus 6-months prednisone therapy during the first episode of NS in patients <4-yr-old [CTRI2015/06/005939; NCT03141970]. Methods: Following ethics approval & parental consent, 172 consecutive patients (1-4 yr-old) were enrolled at onset of idiopathic NS during 2015-19. After 6-wk daily & 6-wk alternate day (AD) initial prednisone therapy, they were randomized (1:1) to either tapering prednisone on AD for 12-wk or no therapy. Relapses were treated with prednisone 2 mg/kg/d till remission, then on AD for 4-wk. Outcomes, based on intention-to-treat analysis during 2-yr follow up, include the proportion of patients with relapse or frequent relapses, time to first relapse, cumulative steroid dose & adverse effects. Based on a prior RCT, at 80% power & α=0.05) 78 patients were required per group to show 30% higher sustained remission with 6-mo therapy. Results: Baseline features in the groups were similar (Fig 1). Despite trends favoring 6-mo therapy, proportions of patients in sustained remission & frequent relapses at 1- & 2-yr, time to relapse (HR 0.75; 95%CI 0.53-1.06) or frequent relapses (HR 0.78; 0.52-1.18), and relapse rates were similar (Fig 1, 2). The rates of adverse events were similar.Fig 1.: Baseline characteristics & key outcome variablesConclusions: Prolonged initial prednisolone therapy does not significantly alter the disease course in young children with NS. Funding: Government Support - Non-U.S.Fig 2.: Kaplan-Meier estimates of the time to (a) relapse, and (b) frequent relapses
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.
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