DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome, type 12 — 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 12 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 12 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
bone morphogenetic protein 7 (BMP7) — BMP7 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 1LXI · 2.0 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
In children with nephrotic syndrome, the majority of cases are minimal change disease, and morbidity and occasional mortality stem from complications such as infections, hypovolaemia, hypercoagulability, hyperlipidaemia, anaemia, and growth delays rather than from renal failure itself. A 2021 randomised controlled trial enrolled 172 children aged 1–4 years with a first episode of idiopathic nephrotic syndrome. After an initial six weeks of daily prednisone followed by six weeks of alternate-day prednisone, patients were randomised to either tapering prednisone on alternate days for another 12 weeks or no further therapy. Over two years of follow-up, the proportion of patients in sustained remission, the rate of frequent relapses, and the time to first relapse were similar between the three-month and six-month groups. The hazard ratio for relapse was 0.75 (95% CI 0.53–1.06) and for frequent relapses was 0.78 (95% CI 0.52–1.18). Adverse event rates were also similar. The authors concluded that prolonged initial prednisolone therapy does not significantly alter the disease course in young children.
A 2025 case report describes a nine-year-old child with nephrotic syndrome who had been on long-term corticosteroids. After three months of an unspecified ayurvedic polyherbal regimen, steroid dependency was reduced; after a further nine months, oedema resolved and urine albumin became nil. The child was followed for one year after treatment completion with no relapse reported. This is a single case, not a controlled trial, and no details of the herbal formulation, dosing, or concurrent steroid tapering schedule are provided.
No randomised evidence supports any specific drug repurposing for nephrotic syndrome type 12. The 2021 trial shows that extending initial prednisone therapy beyond three months does not improve outcomes in young children. The 2025 case report is anecdotal and cannot be generalised. What is missing are trials that stratify patients by genetic subtype, funding for adequately powered studies in steroid-resistant or frequently relapsing populations, and prospective evaluation of any non-corticosteroid agent in a controlled design.
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 International · 2024 · 32 citations · open access
An international, multi-center study evaluated rituximab therapy in childhood steroid-resistant nephrotic syndrome
AbstractThe efficacy and safety of rituximab in childhood steroid-resistant nephrotic syndrome (SRNS) remains unclear. Therefore, we conducted a retrospective cohort study at 28 pediatric nephrology centers from 19 countries in Asia, Europe, North America and Oceania to evaluate this. Children with SRNS treated with rituximab were analyzed according to the duration of calcineurin inhibitors (CNIs) treatment before rituximab [6 months or more (CNI-resistant) and under 6 months]. Primary outcome was complete/partial remission (CR/PR) as defined by IPNA/KDIGO guidelines. Secondary outcomes included kidney failure and adverse events. Two-hundred-forty-six children (mean age, 6.9 years; 136 boys; 57% focal segmental glomerulosclerosis, FSGS) were followed a median of 32.4 months after rituximab. All patients were in non-remission before rituximab. (146 and 100 children received CNIs for 6 month or more or under 6 months before rituximab, respectively). In patients with CNI-resistant SRNS, the remission rates (CR/PR) at 3-, 6-, 12- and 24-months were 26% (95% confidence interval 19.3-34.1), 35.6% (28.0-44.0), 35.1% (27.2-43.8) and 39.1% (29.2-49.9), respectively. Twenty-five patients were in PR at 12-months, of which 22 had over 50% reduction in proteinuria from baseline. The remission rates among children treated with CNIs under 6 months before rituximab were 42% (32.3-52.3), 52% (41.8-62.0), 54% (44.3-64.5) and 60% (47.6-71.3) at 3-, 6-, 12-, and 24-months. Upon Kaplan-Meier analysis, non-remission and PR at 12-months after rituximab, compared to CR, were associated with significantly worse kidney survival. Adverse events occurred in 30.5% and most were mild. Thus, rituximab enhances remission in a subset of children with SRNS, is generally safe and CR following rituximab is associated with favorable kidney outcome.
Rossiyskiy Vestnik Perinatologii i Pediatrii (Russian Bulletin of Perinatology and Pediatrics) · 2017 · 13 citations · open access
NEPHROTIC SYNDROME: PAST, PRESENT AND FUTURE
AbstractThis literature review is focused to change our ideas about the etiology, pathogenesis and treatment tactics of the nephrotic syndrome in recent decades. The change in the treatment outcomes of the primary nephrotic syndrome in connection with the emergence of new therapy technologies, is shown. Features of the course, examination and therapy of congenital and infantile nephrotic syndrome and the possibility of the debut of a nephrotic syndrome associated with various gene mutations and at an older age are presented. Principal differences in diagnostic and therapeutic approaches are accentuated depending on the cause of the development of the disease. Modern syndromological and pathogenetic methods of therapy of primary nephrotic syndrome are presented, and the immediate opportunities for the introduction of new treatment technologies based on the use of monoclonal antibodies, are shown.
Immunosuppressive treatment of idiopathic membranous nephropathy: the dilemma continues
AbstractIdiopathic membranous nephropathy(IMN) is one of the most common causes of nephrotic syndrome (NS) in adults and may progress to end-stage renal disease(ESRD). Given the variable course, it remains unclear who to treat with immunosuppression(IS) and with what regimen. Corticosteroids, alkylating agents, calcineurin inhibitors (CNIs), and antimetabolities have all been used in randomized controlled trials (RCTs). Previous meta-analyses of these trials were unable to demonstrate a benefit on death or progression to ESRD compared to no treatment or placebo. Since the last round of these analyses (in 2004) additional RCTs have been published. The Cochrane Central Register of Controlled Trials and Medline were searched from 2003 until February 2012 for new RCTs in the treatment of IMN to update the database. Twelve trials were found. Due to significant heterogeneity of patients and regimens, they are discussed qualitatively only and are integrated with prior RCTs and relevant observational data. In conclusion, patients with non-nephrotic proteinuria should not be offered IS therapy. Those with NS and declining renal function should be treated. The best evidence supports a combined steroid and alkylating agent regimen. Calcineurin inhibitors clearly produce short-term benefit (proteinuria reduction and remission) but their ability to favorably affect death or ESRD remains unproven. There is little support for antimetabolite use. Other agents (rituximab and adrenocorticotropin) require further study. For the large group of patients with NS but normal renal function it remains a dilemma who to treat and with regimen.
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.
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
Kidney International Reports · 2019 · 1 citations · open access
MON-028 PATHOLOGICALLY PROVEN COMPLETE CURE OF IDIOPATHIC MEMBRANOUS GLOMERULONEPHRITIS BY METHYLPREDNISOLONE PULSE THERAPY
AbstractMGN is the second most common cause of nephrotic syndrome in adults, with FSGS recently becoming the most common, however there is no cure for idiopathic MGN and treatment focus on conservative methods such as salt restriction, blood thinning agents, ARB etc. Sometimes corticosteroids, cyclophosphamide, chlorambucil, cyclosporine-A, tacrolimus, MMF, Rituximab etc are tried with mixed results.
Annals of Ayurvedic Medicine · 2025 · 0 citations · open access
Ayurvedic Modalities in the Management of Nephrotic Syndrome: A Case Study
AbstractNephrotic syndrome is a disorder having symptoms of high levels of protein excretion in the urine (more than 40 mg/m2 per hour), gravity-dependent edema, hypoalbuminemia, hyperlipidemia, and in certain cases, hypertension. With an incidence of around 2 to 3 cases per 100,000 children, it is mostly encountered in the pediatric population. Causes can be Idiopathic, congenital, or secondary. It is mostly idiopathic in children and is frequently referred to as primary nephrotic syndrome. Up to 20% of ESRD and 12% of chronic renal disease are caused by primary nephrotic syndrome. To achieve remission, patients often require corticosteroids, although many either relapse after remission or are unresponsive to treatment. The better alternative is, however far from established. In the treatment of steroid-dependent or resistant cases of Nephrotic Syndrome, polyherbal formulations with an immunomodulator, nephroprotective, and antioxidant activity may be more efficient than modern therapeutic drugs. We present a case of nephrotic syndrome in a 9-year-old child who was taking corticosteroids for a long time & after 03 months of ayurvedic treatment, we diminished the dependency on steroids, and after another 09 months, the edema was relieved and urine albumin levels became Nil. The child was followed up for 1 year after treatment completion and there was no relapse of the disease noted. .
Tropical Journal of Medical Research · 2011 · 0 citations · open access
Nephro-Prevention: An old concept to prevent childhood renal disorder
AbstractTwo children with renal disorders managed at the Aminu Kano Teaching Hospital, Kano are reported to show the importance of nephro-prevention.The first report of a rapidly progressive nephropathy who failed to assess care early due to financial constraints. Subsequent efforts to help the childwere inadequate and the child was discharged against medical advice. The second case represents an important and common group of renal disorderwith relapsing type of nephrotic syndrome, which is treatable. All it entails is long term follow-up and attention to factors that may lead to relapse. Inthe two cases, primary, secondary and tertiary preventive strategies were useful.
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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