DeCure's autonomous Nephrology AI scientist is researching a drug-repurposing hypothesis for nephrotic syndrome, type 8 — 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 8 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 8 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
Rho GDP dissociation inhibitor alpha (ARHGDIA) — ARHGDIA 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 gdpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 1HH4 · 2.7 Å · ligand GUANOSINE-5'-DIPHOSPHATE (GDP). Experimental structure, not a prediction.
What the evidence adds up to
A 2024 systematic review of 11 articles on steroid-sensitive nephrotic syndrome relapse in children found that age, gender, serum creatinine, and serum protein are not risk factors. The review reported conflicting results across studies for proposed risk factors such as hematuria, hypertension, time from treatment to response, and number of relapses. The authors concluded that no conclusion can be reached due to heterogeneity of study designs.
A 2022 Bayesian network analysis examined immunosuppressive agents for refractory nephrotic syndrome in adults, aiming to clarify efficacy and acceptability at inducing remission and managing adverse reactions. The abstract provides no numerical results, no sample size, and no comparison between agents.
A 2019 study funded by Kidney Research UK investigated why a faulty gene involved in nephrotic syndrome leads to disease in some patients. The findings were described as revealing new targets to intervene in the process, but no specific drug, mechanism, or patient data are reported. Around 1 in 50,000 children are diagnosed with nephrotic syndrome each year.
No drug is named in any of these abstracts. No concrete survival or response rates are given. What is missing is standardised trial designs for paediatric relapse risk, head-to-head adult immunosuppressant trials with reported outcomes, and any validated biomarker or genetic stratification that could guide treatment.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
PEDIATRICS · 1959 · 9 citations
TREATMENT OF THE NEPHROTIC SYNDROME WITH TRIAMCINOLONE
AbstractIn this report, only acute remissions of the nephrotic syndrome following triamcinolone therapy are considered. No conclusions are drawn regarding the effects of this treatment on the ultimate fate of these children. With these reservations kept clearly in mind, this experience with 15 children with the nephrotic syndrome indicates that triamcinolone is a highly effective therapeutic agent. With the 20 mg/day dose employed for 30-45 days in this series, side-effects were absent except for the occasional appearance of slight moon facies. The incidence of complete remission in the entire series was 10 out of 15 patients (67%). Of patients whose disease was less than 12 months in duration, and who did not have hypertension, eight out of nine patients (89%) had a complete remission. These results compare favorably with the experience reported in the literature with other steroids. The mere induction of acute remissions, no matter how readily or reproducibly, should not be the chief concern in therapy of nephrosis. The ultimate prognosis is still pessimistic, despite regimens of therapy with currently available steroids. A recent report suggests that the long-term results of steroid therapy in the nephrotic syndrome might be improved by earlier, more intensive, and more prolonged treatment. It is the present authors impression that triamcinolone is a useful steroid for such a program, because of its high potency and minimal side-effects. The doses employed did not cause hypertension or retention of sodium.
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.
Kidney International Reports · 2022 · 0 citations · open access
POS-218 Immunosuppressive Agents for Refractory Nephrotic Syndrome in adults: A Bayesian Network Analysis
AbstractNephrotic syndrome is a common and frequently occurring disease in chronic kidney diseases. However, some patients are often difficult to cure due to frequent recurrence, and can lead to unpredictable complications, side effects of steroids and immunosuppressants, and even life-threatening. Therefore, this study aimed to clarify the efficacy and acceptability of immunosuppressive therapy at inducing remission and adverse reaction in refractory nephrotic syndrome (RNS).
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.
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.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
