DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome 15 — 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 15 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 15 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 a cohort of 91 children with nephrotic syndrome, 82.4% had steroid-sensitive disease and 17.6% were steroid-resistant. Among the steroid-sensitive group, 56% were over six years old and 68% were male. Risk factors for relapse identified in a separate study of 100 children with steroid-sensitive nephrotic syndrome included infection during the initial attack and first relapse, a shorter time between remission in the first episode and the first relapse, inadequate treatment duration, lower serum albumin, and higher cholesterol.
A single-centre prospective study compared mycophenolate mofetil (MMF) and tacrolimus (TAC) in 72 children with frequently relapsing or steroid-dependent nephrotic syndrome. In the MMF group (n=34), the mean six-month relapse rate fell from 2.56 episodes before therapy to 0.76 in the first six months and 0.67 in the second six months. In the TAC group (n=38), the rate fell from 2.39 to 0.41 and then 0.42. There was no significant difference between the two drugs in relapse rate, cumulative sustained remission, or adverse events. Both were described as a promising strategy with a moderate risk of side effects when combined with low-dose steroids.
The underlying mechanisms of primary nephrotic syndrome remain unclear, though immunity — cellular, humoral, and immunity involving podocytes — is thought to play a role. A faulty gene linked to nephrotic syndrome has been identified, and research has provided new insights into why it leads to disease in some patients, suggesting potential targets for intervention. Around 1 in 50,000 children are diagnosed with nephrotic syndrome each year.
What is still missing is a molecular approach that accounts for the heterogeneity of the disease, as the clinical presentation can be homogeneous while the underlying causes are diverse. No trial has yet tested whether stratifying patients by genetic or immune markers improves outcomes. The studies cited are single-centre and relatively small; larger, multi-centre trials with longer follow-up and standardised definitions of relapse and remission are needed. Funding for such trials and for translational work linking genetic findings to treatment decisions remains limited.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Pediatric Transplantation · 2004 · 33 citations · open access
The pediatric nephrotic syndrome spectrum: Clinical homogeneity and molecular heterogeneity
AbstractIdiopathic nephrotic syndrome is the most common glomerular disorder of childhood. Recurrence of nephrotic syndrome immediately following renal transplantation is rapid, results in a high rate of graft loss, and represents the most severe form of nephrotic syndrome. This review discusses the molecular heterogeneity of pediatric nephrotic syndrome across the spectrum of disease activity. A schema is offered for a molecular approach to pediatric nephrotic syndrome, including immune-mediated and structural/genetic factors.
Evaluation of mycophenolate mofetil or tacrolimus in children with steroid sensitive but frequently relapsing or steroid‐dependent nephrotic syndrome
AbstractAIM: Approximately 30-40% of children with steroid sensitive nephrotic syndrome have frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS). Mycophenolate mofetil (MMF) and tacrolimus (TAC) are often alternative treatment choices for these patients. METHODS: A single-center prospective study was conducted to compare the efficacy of MMF or TAC in reducing relapses and maintaining remission in children with FRNS or SDNS. Of the 72 recruited patients, either MMF (20∼30 mg/kg/d, n = 34) or TAC (0.05∼0.15 mg/kg/d, n = 38) was administered for 12 months. RESULTS: The mean 6-month relapse rates decreased from 2.56 episodes before therapy to 0.76 episodes in the first 6 months after therapy (c(2) = 44.362, p < 0.001) and 0.67 in the next 6 months (c(2) = 37.817, p < 0.001) in the MMF group. In the TAC group, the mean 6-month relapse rates decreased from 2.39 episodes before therapy to 0.41 episodes in the first 6 months after therapy (c(2) = 62.242, p < 0.001) and 0.42 in next 6 months (c(2) = 67.482, p < 0.001). No significant difference in the relapse rate was found between the groups (before therapy, c(2) = 0.902, p = 0.637; first 6 months, c(2) = 5.358, p = 0.147; second 6 months, c(2) = 4.089, p = 0.252). And there was also no significant difference in cumulative sustained remission and the incidence of adverse events between two groups. CONCLUSIONS: In combination with low-dose steroids, MMF or TAC presented similar efficacy in maintaining remission in children with FRNS/SDNS in the present study. Therapy with MMF or TAC is a promising strategy with a moderate risk of side effects in children who are steroid sensitive but have FRNS/SDNS.
AbstractSuccinct aspects of clinical features, pathophysiology and prognosis of nephrotic syndrome in childhood, and indications for performing renal biopsy are enumerated in this review. Orthodox treatment of the nephrotic syndrome with more recent therapeutic approaches, and the role of diuretics, albumin infusions and immunizations in patients with the nephrotic syndrome are reviewed. The etiology of peritonitis, acute renal failure, and renal transplantation are re-examined to update nephrologists on the associated complications of this common childhood disease.
Levamisole in steroid dependent and frequently relapsing nephrotic syndrome.
AbstractOBJECTIVE: To determine the efficacy of levamisole in steroid dependent (S.D) and frequently relapsing (F.R) nephrotic, from syndrome (N.S). DESIGN: Quasi-experimental study. PLACE AND DURATION OF STUDY: Department of Nephrology at The Children s Hospital, Lahore, over a period of 5 years from January 2000 to December 2004. MATERIAL AND METHODS: S.D.N.S and F.R.N.S patients between the ages of 1-15 years, were given levamisole on alternate day in a dose of 2.5 mg/kg, if either the dosage of steroids to maintain remission was >1 mg/kg/every other day (EOD), or 0.5 mg/kg/EOD with signs of steroid toxicity. The agent was continued for a period of one year and the steroids were gradually tapered off by 2.5-5 mg every four weeks to less than 0.5 mg/kg/EOD. The patients were monitored for maintenance of remission and side effects of drug. RESULTS: Seventy patients with a mean age of 5.50+/-2.97 years , with male to female ratio of 4:1 were studied. Nineteen (27.14%) patients did not relapse on therapy, while it was ineffective in 11(15.7%). Rest of 40 (57.14%) patients, though, relapsed during therapy, their duration of remission was prolonged from six months to one year, and dose of corticosteroids could be significantly reduced (0.1-0.3 mg/kg/EOD). It was also observed that levamisole is more effective in older children (>5 years versus <5 years) [P-value 0.03]. The only side effects were transient rash and occasional vomiting. CONCLUSION: Levamisole is a safe and effective steroid sparing drug, in steroid dependent and frequently relapsing nephrotic syndrome, for the prolongation of remission, especially in older children.
OUTCOME SINDROM NEFROTIK PADA ANAK – PENELITIAN PROSPEKTIF STUDI COHORT
AbstractNephrotic syndrome (NS) in children is a common recurrent disease. Most of the cases are due to minimal change disease with a favorable outcome. The mayority of children have minimal change disease and 90 – 95% will respond to steroid therapy. Response to steroid therapy carries a greater prognostic weightthan the histologic features. The aim of the studywas to describe the outcome of Nephrotic Syndrome in children and to determine risk factors for these complications. Children with NSwere admitted to Pediatric Department Saiful Anwar Hospital Malang, January 2000 - December 2003 evaluated prospectively for one year. Data was sought on steroid responsiveness, remission, relapse rates, infection, and trombosis. Patients were classified into five categories. Including: relapse, infrequent relapsing (IFRNS), frequent relapsing (FRNS), steroid dependent (SDNS) and steroidresistant (SRNS). Baseline age, gender, clinical manifestation and laboratory finding were used to predict category of the disease. Definition of NS, remission and relapse were based on the ISKDC guidelines. Of 91 children with NS, 75 (82,4%) children had steroid sensitive nephrotic syndrome (SSNS) while 16 (17,6%) children were classified SRNS. In the SSNS group 42 (56%) of children were over 6 years of age and there were 51 (68%) males and 24 (32%) females.
Journal of Family Medicine and Primary Care · 2023 · 1 citations · open access
Risk factors for relapse in pediatric nephrotic syndrome in Ranchi
AbstractIntroduction: The nephrotic syndrome (NS) is a common childhood illness characterized by massive proteinuria, hyperlipidemia, and hypoalbuminemia. It is a disease of relapse, and therefore, it is a major problem to manage cases with frequent relapses. Prediction and prevention of risk factors is the key to successful management of childhood NS. An understanding of the risk factors that determine the course is useful in taking decisions regarding therapy and enables counseling. Materials and Methods: Sample size of 100 children of age 1-12 years of age with steroid sensitive nephrotic syndrome over duration of 1 year from April 2020 to May 2021. Results and Conclusion: Risk factors for relapse were presence of infection during initial attack and first relapse as well as less time interval between remission in first episode of nephrotic syndrome and first relapse.incresed risk was also associated with inadequate treatment duration and less serum albumin level and high cholesterol level.
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.
International journal of pediatrics · 2016 · 0 citations
Advance in immunopathogenesis of primary nephrotic syndrome
AbstractPrimary nephrotic syndrome(PNS) is a common kidney disease.However, its mechanisms remain unclear, and immunity might to play an important role in PNS.This article will review the pathology from cellular immunity, humoral immunity and the immunity involved in podocytes.It is useful for further understanding, and it may help guide the diagnosis, prognosis and therapeutic strategies.
Key words:
Primary nephrotic syndrome; Immunity; Pathogenesis; Children
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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