DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome 14 — 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 14 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 14 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
sphingosine-1-phosphate lyase 1 (SGPL1) — SGPL1 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 sindrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4Q6R · 2.4 Å · ligand SUCCINIC ACID (SIN). Experimental structure, not a prediction.
What the evidence adds up to
A 2017 case report describes a two-year-old boy with frequently relapsing nephrotic syndrome whose relapses were suppressed by azithromycin, allowing him to avoid long-term immunosuppressive therapy. The authors suggest azithromycin could be a new treatment option for selected cases, but this is a single case, not a controlled trial. No other abstracts test azithromycin or any other drug for this condition.
A 2013 prospective cohort study of 91 children with nephrotic syndrome in Indonesia found that 75 (82.4%) had steroid-sensitive disease and 16 (17.6%) were steroid-resistant. Among the steroid-sensitive group, 42 (56%) were over six years old, and 51 (68%) were male. The study describes outcomes but does not test any new treatment.
A 2024 systematic review of risk factors for relapse in steroid-sensitive nephrotic syndrome in children analysed 11 studies and found that age, gender, serum creatinine, and serum protein were not risk factors. Possible associations were reported for hematuria, hypertension, time from treatment to response, and number of relapses, but the authors state that results were conflicting across studies and that no conclusion can be reached due to heterogeneity. Two other abstracts from 2017 and 2019 review the role of cytokines such as interleukins and interferon-gamma in the pathogenesis of primary nephrotic syndrome, but neither reports clinical trial data or treatment outcomes.
What is still missing is any randomised controlled trial of azithromycin or any other repurposed drug for nephrotic syndrome relapse. The single case report cannot support a treatment recommendation. The systematic review shows that even basic risk factors for relapse remain poorly understood due to inconsistent study designs. No trial funding, no standardised patient stratification, and no prospective comparative data exist for the proposed use of azithromycin.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Azithromycin suppressed relapses of idiopathic nephrotic syndrome in a child
AbstractLong-term immunosuppressive therapy with severe adverse effects is indispensable to maintain disease remission in frequently relapsing nephrotic syndrome (NS) in children. Hence, development of new therapy with less toxicity for relapses of NS is required. We demonstrated a case of a 2-year-old boy with frequently relapsing NS, whose frequent relapses were successfully treated with azithromycin. Azithromycin treatment prevented the need for long-term immunosuppressive therapy in this case. Azithromycin could be a new treatment option for relapse of NS, with few adverse effects, in selected cases.
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.
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 Pediatrics Review · 2024 · 1 citations · open access
A Systematic Review on the Risk Factors of Steroid-Sensitive Nephrotic Syndrome Relapse in the Pediatric Population
AbstractRisk Factors of Steroid-sensitive Nephrotic Syndrome Relapse in the Pediatric Population Background: Identifying affecting and predictive factors of steroid-sensitive nephrotic syndrome's (SSNS's) outcome may greatly benefit the proper management of SSNS patients. Objectives:The current systematic review comprehensively reviews all available evidence on the risk factors of SSNS relapse in children and adolescents.Methods: An extensive search was conducted on the electronic databases of Medline, Embase, Web of Science, and Scopus until February 18, 2024.Studies investigating the risk factors of relapse were included in this systematic review.Results: A total of 11 articles were included.Age, gender, and laboratory variables, such as serum creatinine and serum protein are not risk factors for relapse in these studies.Possible associations were reported for risk factors, such as the number of relapses and response time.Overall, the studies reported conflicting results on the value of relapse risk factors. Conclusions:Although factors, such as hematuria, hypertension, time from treatment to response, and number of relapses have been proposed as possible risk factors for relapse, no conclusion can be reached due to the heterogeneity of studies.Future studies should have more conforming designs to make comparisons more reliable.
International journal of pediatrics · 2019 · 0 citations
Research and progress of cytokines in pathogenesis of primary nephrotic syndrome
AbstractPrimary nephrotic syndrome(PNS)is a common renal disease in children and the pathogenesis has not been clarified, but it is considered to be related to cellular immune dysfunction.We collected the reported cytokines associated with PNS and reviewed the resources and the involving pathogenesis of the cytokines.We hope to help to clarify the possible pathogenesis of PNS and to develop new treatments for PNS patients.
Key words:
Primary nephrotic syndrome; Cytokine; T-lymphocyte; Interleukin
International journal of pediatrics · 2017 · 0 citations
Role of IL-21 and INF-γ in the pathogenesis of nephrotic syndrome
AbstractThe etiology and pathogenesis of nephrotic syndrome is not fully clear.Most pepole believe that it is associated with the disorder of immune function.As immunoregulatory factors, interleukins(IL) and interferon(IFN) are closely related with the onset of nephrotic syndrome.In this paper, we review the role of IL-21 and INF-γ in pathogenesis of nephrotic syndrome.
Key words:
Nephrotic syndrome; Interleukin-21; Interferon-γ
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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