DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome, type 11 — 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 11 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 11 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.
What the evidence adds up to
In acquired nephrotic syndrome, glomerular nephrin mRNA levels are significantly decreased in minimal change disease, with an apparent reduction also seen in a single case of membranous nephropathy, based on a 1999 study of four nephrotic syndrome cases (three minimal change, one membranous) and six normal controls. The authors concluded that abnormalities of nephrin expression appear associated with acquired as well as congenital causes of nephrotic syndrome.
A 2008 Cochrane review of interventions for minimal change disease in adults identified only three randomised controlled trials (68 participants total), each comparing different regimens. No significant difference was found between prednisone and placebo for complete remission (RR 1.44, CI 0.95 to 2.19) or partial remission (RR 1.00, CI 0.07 to 14.45). There was no difference between intravenous methylprednisolone plus oral prednisone compared with oral prednisone alone for complete remission (RR 0.74, CI 0.50 to 1.08). Prednisone compared with short-course intravenous methylprednisolone increased the number achieving complete remission (RR 4.95, CI 1.15 to 21.26), but the authors noted the lack of statistical evidence for prednisone efficacy was based on a single small study. No RCTs were identified for steroid-dependent or relapsing disease, or for alkylating agents, cyclosporine, tacrolimus, levamisole, or mycophenolate mofetil.
A 2021 randomised controlled trial in 172 children aged 1–4 years with first-episode idiopathic nephrotic syndrome compared 3 months versus 6 months of initial prednisone therapy. Despite trends favouring 6-month therapy, the proportions of patients in sustained remission and frequent relapses at 1 and 2 years, time to relapse (HR 0.75; 95%CI 0.53–1.06), time to frequent relapses (HR 0.78; 0.52–1.18), and relapse rates were similar between groups. 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 study describes a 9-year-old child with nephrotic syndrome on long-term corticosteroids who, after 3 months of ayurvedic treatment, had diminished steroid dependency, and after a further 9 months had no edema and nil urine albumin. The child was followed for one year after treatment completion with no relapse reported. This is a single case report with no control group, no blinding, and no replication. What remains missing are adequately powered randomised trials comparing ayurvedic regimens to standard care, trials that stratify patients by steroid responsiveness or genetic subtype, and funding for such studies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Glomerular expression of nephrin is decreased in acquired human nephrotic syndrome
AbstractBACKGROUND: Nephrin recently has been identified as a putative adhesion molecule, expressed in the glomerulus, in which mutations cause congenital nephrotic syndrome of Finnish type. We sought to determine whether expression of nephrin is altered in human glomeruli in patients with acquired nephrotic syndrome. METHODS: We performed PCR amplification of nephrin cDNA, using cDNA previously prepared from single human glomeruli plucked fresh from the surface of human renal biopsies. We had available four cases of nephrotic syndrome (one membranous, three minimal change) and six normal controls. PCR product quantitation was by gel densitometry, confirmed by enzyme-linked immunosorbent assay using a specific oligonucleotide probe. Results were corrected for reaction efficiency and glomerular cellularity by expression as a ratio to levels of the 'housekeeping gene' glyceraldehyde phosphate dehydrogenase. RESULTS: Glomerular levels ofnephrin mRNA are significantly decreased in cases of minimal change nephrotic syndrome. An apparent reduction was also seen in the single case of membranous nephropathy which was available for study. CONCLUSIONS: Abnormalities of nephrin expression appear to be associated with acquired as well as congenital causes of human nephrotic syndrome.
Cochrane Database of Systematic Reviews · 2008 · 47 citations · open access
Interventions for minimal change disease in adults with nephrotic syndrome
AbstractBACKGROUND: Steroids have been used widely since the early 1970s for the treatment of adult-onset minimal change disease. The response rates to immunosuppressive agents in adult minimal change disease, especially steroids, are more variable than in children. The optimal agent, dose, and duration of treatment for the first episode of nephrotic syndrome, or for disease relapse(s) has not been determined. OBJECTIVES: To determine the benefits and harms of interventions for the nephrotic syndrome in adults caused by minimal change disease. SEARCH STRATEGY: We searched the Cochrane Central Register of Controlled Trials, MEDLINE, EMBASE, reference articles and abstracts from conference proceedings, without language restriction. Search date: January 2007. SELECTION CRITERIA: Randomised controlled trials (RCTs) and quasi-RCTs of any intervention for minimal change disease in adults over 18 years with the nephrotic syndrome were included. Studies comparing different routes, frequencies, and duration of immunosuppressive agents were selected. Studies comparing non-immunosuppressive agents were also assessed. DATA COLLECTION AND ANALYSIS: Two authors independently assessed study quality and extracted data. Statistical analyses were performed using the random effects model and results were expressed as a relative risk (RR) for dichotomous outcomes, or mean difference (WMD) for continuous data with 95% confidence intervals (CI). MAIN RESULTS: Three RCTs (68 participants) were identified. All treatment comparisons contained only one study. No significant difference was found between prednisone compared with placebo for complete (RR 1.44, CI 0.95 to 2.19) and partial remission (RR 1.00, CI 0.07 to 14.45) of the nephrotic syndrome due to minimal change disease. There was no difference between intravenous methylprednisolone plus oral prednisone compared with oral prednisone alone for complete remission (RR 0.74, CI 0.50 to 1.08). Prednisone, compared with short-course intravenous methylprednisolone, increased the number of subjects who achieved complete remission (RR 4.95, CI 1.15 to 21.26). The lack of statistical evidence of efficacy associated with prednisone therapy was based on data derived from a single study that compared 'alternate-day prednisone' to no immunosuppression' with only a small number of participants in each group. No RCTs were identified comparing regimens in adults with a steroid-dependent or relapsing disease course or comparing treatments comprising alkylating agents, cyclosporine, tacrolimus, levamisole, or mycophenolate mofetil. AUTHORS' CONCLUSIONS: Further comparative studies are required to examine the efficacy of immunosuppressive agents for achievement of sustained remission of nephrotic syndrome caused by minimal change disease. Studies are also needed to evaluate treatments for adults with steroid-dependent or relapsing disease.
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
Brazilian Journal of Nephrology · 2022 · 1 citations · open access
Outcomes of children with idiopathic steroid resistant nephrotic syndrome: a single centre observational study
AbstractINTRODUCTION: Idiopathic steroid resistant nephrotic syndrome (SRNS) has variable outcomes in children. The primary objective of the present study was to assess the cumulative remission rate and the secondary objectives were to assess factors affecting the remission status, kidney function survival, and adverse effects of medications. METHODS: One hundred fourteen patients with SRNS were included. Calcineurin inhibitor-based treatment protocol along with prednisolone and angiotensin-converting enzyme inhibitor were used, and patients were followed over 5 years. RESULTS: Median age was 4.5 years; 53.5% of cases were between 1 to 5 years of age. Sixty-two patients (54.4%) were at initial stage and 52 (45.6%) were at a late SRNS stage. Median eGFRcr was 83.5 mL/min/1.73m2 at presentation. Of the 110 patients, 63 (57.3%) achieved remission [complete remission 30 (27.3%), partial remission 33 (30%)], and 47 (42.7%) had no remission. Kidney function survival was 87.3% and 14 cases (12.7%) had progression to CKD (G3-8, G4-3, G5-1, and G5D-2). Median duration of follow up was 36 months (IQR 24, 60). Age of onset, cyclosporine/tacrolimus, eGFRcr, and histopathology (MCD/FSGS) did not affect remission. Similarly, remission status in addition to age of onset, drug protocol, and histopathology did not significantly affect kidney function during a period of 5 years. Hypertension, cushingoid facies, short stature, cataract, and obesity were observed in 37.7, 29.8, 25.5, 17.5, and 0.7% of cases, respectively. CONCLUSION: About half of the cases achieved remission. Age of onset of disease, cyclosporine/tacrolimus use, and histopathological lesion neither affected remission status nor short-term kidney function survival in SRNS.
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. .
Kidney International Reports · 2020 · 0 citations · open access
SUN-372 CD 19 targeted Rituximab is safe and effective in the management of Steroid dependent/resistant Podocytopathy
AbstractMinimal change disease (MCD) and focal segmental glomerulosclerosis (FSGS) are common podocytopathies causing nephrotic syndrome (NS) in adults. A significant proportion of patients are either steroid dependent (SD) or resistant (SR), requiring alternate therapies. Long-term calcineurin inhibitors (CNI) use is associated with dyslipidaemia, impaired glucose tolerance and, nephrotoxicity. Rituximab is a potential candidate in this group of patients, with a more favourable safety profile, so the present study was undertaken to evaluate the efficacy and safety of rituximab in SD/SR MCD/FSGS to maintain remission.
Zenodo (CERN European Organization for Nuclear Research) · 2025 · 0 citations · open access
Steroid Responsive Nephrotic Syndrome
AbstractNephrotic syndrome (NS) is a glomerular condition that causes excessive proteinuria, hypoalbuminemia, hyperlipidemia, and peripheral edema. The case of 8 year old male child weighing 24 kg with a one week history of edema, the swelling around the eyes and legs. Examination indicated bilateral pedal edema (pitting kind). Laboratory results revealed protein in urine, low serum albumin, and increased lipid levels. However, clinical and laboratory evidence led to the diagnosis of idiopathic nephrotic syndrome (NS). The patient was mostly treated with Cefotaxime, Prednisolone, and Pantoprazole. Urine I/O charting helps assess intake and output.
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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