DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome, type 4 — screening already-approved drugs against its 1-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 4 maps to a 1-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 4 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
WT1 transcription factor (WT1) — WT1 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6BLW · 1.835 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
Nephrotic syndrome in children is primarily a chronic disease with minimal change histology; morbidity and occasional mortality arise from complications such as infections, hypovolaemia, hypercoagulability, hyperlipidaemia, anaemia, and growth delay, not from renal failure itself. A 2022 randomised controlled trial of 40 children with steroid-sensitive nephrotic syndrome and an infrequently relapsing course compared a low-dose prednisolone regimen (2 mg/kg/day until remission, then 1 mg/kg on alternate days for 4 weeks) with standard therapy (2 mg/kg/day until remission, then 1.5 mg/kg on alternate days for 4 weeks). Median time to remission was 9 days in both groups (P = 0.14). All patients were in remission at end of therapy; at 1 month, 85% of the low-dose group and 90% of the standard group remained in remission (P = 0.32). At 3 months, 60% of the low-dose group and 65% of the standard group were still in remission (P = 0.37). Hazard ratios for relapse at 1, 2, and 3 months were 1.05, 1.08, and 1.13 respectively. Among the 79% of children who had been infrequently relapsing from onset, 3-month remission rates were 80% in the low-dose group versus 76.9% in the standard group, with hazard ratios of 1.01, 1.03, and 1.08 at 1, 2, and 3 months.
A 2019 research summary notes that a faulty gene involved in nephrotic syndrome leads to disease in some patients, and that understanding this mechanism might reveal new targets for intervention. The abstract provides no specific gene, no patient data, and no experimental results. Approximately 1 in 50,000 children are diagnosed with nephrotic syndrome each year.
The 2007 review states that untreated nephrotic syndrome can cause multiple complications but gives no quantitative outcomes. The 2022 trial is small (40 children) and limited to steroid-sensitive, infrequently relapsing cases; it does not address steroid-resistant nephrotic syndrome, type 4 nephrotic syndrome, or any genetic subtype. What is still missing is a trial specifically in nephrotic syndrome type 4 (which is typically steroid-resistant and caused by mutations in WT1 or other genes), adequate funding for such a trial, and patient stratification by genotype. No drug other than prednisolone is mentioned in these abstracts.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Apollo Medicine · 2007 · 2 citations
Complications of Nephrotic Syndrome in Children
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 Nephrology · 2022 · 1 citations · open access
Effectiveness of a low dose prednisolone regimen for treatment of relapses in children with steroid sensitive nephrotic syndrome
AbstractIntroduction: There may be a role of reducing the total steroid doses for the treatment of relapses of nephrotic syndrome in children with milder and more stable disease. The primary objective of this study was to compare the effectiveness of a low-dose prednisolone regimen with standard therapy for the treatment of relapses in steroid-sensitive nephrotic syndrome (SSNS) at the end of treatment, the secondary objectives being time to remission and sustained remission after 3 months. Methods: This randomized controlled trial included a total of 40 children (20 in each group) with SSNS (presently infrequently relapsing course) and with a relapse. Both groups received prednisolone at a dose of 2 mg/kg/day until remission; subsequently, the patients in the study group received 1 mg/kg, and the control group participants received 1.5 mg/kg prednisolone on alternate days for 4 weeks. The patients were followed up till 3 months after stopping the therapy. Results: The median (IQR) age of children enrolled was 7.5 (range: 5–9.65) years, and the age at onset of nephrotic syndrome was 4 (range: 2.3–5.5) years. The median time to achieve remission was 9 days (comparable in low dose vs. standard therapy group; P = 0.14). All patients were in remission at the end of therapy; 85% of patients were in the low-dose group and 90% in the standard therapy group after 1 month (P = 0.32). At the end of 3 months, 60% continued to be in remission in the low-dose group and 65% with standard therapy (P = 0.37). Hazard ratios for relapse at the end of 1, 2, and 3 months were 1.05, 1.08, and 1.13, respectively. Patients who were infrequently relapsing (79%) from the onset of nephrotic syndrome had higher remission rates at the end of 3 months (80% in the low-dose group vs. 76.9% in the standard therapy group). Hazard ratios for relapse in these patients at the end of 1, 2, and 3 months were 1.01, 1.03, and 1.08, respectively. Conclusions: Lower doses of prednisolone can be used for the treatment of relapse of steroid sensitive nephrotic syndrome, with an infrequently relapsing course.
Scientists reveal how a faulty gene leads to kidney disease
AbstractResearch funded by Kidney Research UK has provided new insights into why a faulty gene involved in nephrotic syndrome leads to disease in some patients. The findings could pave the way for new ways to prevent or treat the condition, by revealing new targets to intervene in the process. Around 1 in 50,000 children are diagnosed with nephrotic syndrome each year.
Journal of Complementary Medicine Research · 2022 · 0 citations
Research of Approaches to the Treatment of Nephrotic Syndrome
AbstractThe article investigates approaches to the treatment of nephrotic syndrome. Nephrotic syndrome (NS) is one of the main chronic kidney diseases, this disease is diagnosed in patients of any age. At the present stage, there is no universal treatment for this condition. NS therapy currently includes angiotensin converting enzyme inhibitors, angiotensin receptor blockers, monoclonal antibodies, steroids and immunosuppressants. Steroids continue to be the initial approach to the treatment of patients with NS and are considered the basis of therapy. In this regard, it is necessary to consider various innovative approaches to the treatment of this disease in order to increase the effectiveness of NS therapy.
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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