DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Granulomatosis with Polyangiitis — screening already-approved drugs against its 11-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleGranulomatosis with Polyangiitis maps to a 11-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 granulomatosis with polyangiitis 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
myeloperoxidase (MPO) — MPO 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 hemdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5MFA · 1.2 Å · ligand PROTOPORPHYRIN IX CONTAINING FE (HEM). Experimental structure, not a prediction.
What the evidence adds up to
A 2014 case report describes a 32-year-old woman with granulomatosis with polyangiitis who initially presented with progressive, painful oral ulcers and was misdiagnosed with severe infectious necrotising gingivitis. She received combination antibiotics and surgical debridement including extraction of all maxillary and three mandibular teeth before a positive cytoplasmic anti-neutrophil cytoplasmic antibody test and systemic symptoms led to the correct diagnosis. Treatment with rituximab and methylprednisone was chosen because the patient wanted to preserve future fertility, and this regimen was reported as successful in inducing and maintaining remission in that single patient.
A 2019 report on lung-limited granulomatosis with polyangiitis describes a patient who presented with a lung nodule that was diagnosed on excision, had no systemic features, and was ANCA-negative. That patient was managed conservatively without immunosuppression, with the suggestion that such patients be fully informed of the possibility of relapse if close monitoring is chosen. No numbers on relapse rates or long-term outcomes are given.
A 2023 review of pharmacological treatment trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform is mentioned in two supplemental material entries, but no results, drug names, sample sizes, or efficacy data from that review are provided in the available text. The abstracts contain no information on any other drugs, no response rates, no survival data, and no comparative trial outcomes.
What is still missing are large, randomised controlled trials that compare rituximab and methylprednisone against other regimens specifically for fertility-sparing treatment, prospective studies on the natural history of ANCA-negative lung-limited disease with defined relapse rates, and any trial data from the 2023 review that would allow assessment of which pharmacological treatments are effective. The evidence base remains limited to single case reports and a review whose results are not reported here.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Medical Case Reports · 2014 · 10 citations · open access
Granulomatosis with polyangiitis (Wegener’s) as a necrotizing gingivitis mimic: a case report
AbstractINTRODUCTION: Granulomatosis with polyangiitis poses a significant diagnostic dilemma due its diverse presentations. Seemly isolated sites of disease, such as oral ulcers, may present to physicians working in primary care settings, the emergency room, and subspecialty fields as well as to dentists. Oral presentations are particularly challenging to identify and require a high index of suspicion and a detailed knowledge of the condition in order to diagnose and treat. We detail a case of granulomatosis with polyangiitis presenting as necrotizing gingivitis, one of the first of its kind to be reported. CASE PRESENTATION: An otherwise healthy 32-year-old, Caucasian woman presented to various physicians with progressive, painful oral ulcers. Following consultations with multiple primary care physicians and subspecialties, an initial diagnosis of severe infectious necrotizing gingivitis was made resulting in combination antibiotic treatment as well as surgical debridement involving extraction of all maxillary and three mandibular teeth. With the discovery of a positive cytoplasmic anti-neutrophil cytoplasmic antibody and a constellation of associated systemic symptoms, our patient was subsequently diagnosed with granulomatosis with polyangiitis. The treatment regimen of rituximab and methylprednisone was chosen in consideration of our patient's desire for future fertility and has been successful in inducing and maintaining remission. CONCLUSIONS: Following the case presentation, we review the current literature regarding granulomatosis with polyangiitis presentation, diagnosis and treatment. In discussing features of granulomatosis with polyangiitis presentation, diagnostic tests, and important new treatment options, we seek to enable physicians of all specialties to better recognize and begin appropriate treatment for this complex condition.
sj-docx-1-trd-10.1177_26330040231213888 – Supplemental material for The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform
AbstractSupplemental material, sj-docx-1-trd-10.1177_26330040231213888 for The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform by Janet Sultana, Nikita Camilleri, Salvatore Crisafulli, John Joseph Borg, Silvan Spagnol, Silvia Tillati and Joseph Borg in Therapeutic Advances in Rare Disease
Lung-limited granulomatosis with polyangiitis: managed without immunosuppression
AbstractLimited granulomatosis with polyangiitis presenting as a lung nodule, with the diagnosis made on excision, with no systemic features and negative ANCA, may be treated conservatively (without immunosuppression) if closely monitored but it Is suggested that patients are fully informed of the possibility of relapse.
Sage Journals Data · 2023 · 0 citations · open access
sj-docx-1-trd-10.1177_26330040231213888 – Supplemental material for The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform
AbstractSupplemental material, sj-docx-1-trd-10.1177_26330040231213888 for The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform by Janet Sultana, Nikita Camilleri, Salvatore Crisafulli, John Joseph Borg, Silvan Spagnol, Silvia Tillati and Joseph Borg in Therapeutic Advances in Rare Disease
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.
