DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hereditary hemorrhagic telangiectasia — screening already-approved drugs against its 17-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHereditary hemorrhagic telangiectasia maps to a 17-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 hereditary hemorrhagic telangiectasia 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
platelet derived growth factor receptor alpha (PDGFRA) — PDGFRA 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 dimethylaminodrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5GRN · 1.77 Å · ligand N-[2-(dimethylamino)ethyl]-N-[[4-[[4-methyl-3-[(4-pyridin-3-ylpyrimidin-2-yl)amino]phenyl]carbamoyl]phenyl]methyl]pyridine-3-carboxamide (748). Experimental structure, not a prediction.
What the evidence adds up to
Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant vascular condition with variable epidemiology by region. Delays in diagnosis are common because the condition is infrequent, and this can lead to serious complications and poor health outcomes. The Curaçao criteria should be used to assist diagnosis and to identify patients who need further investigations. Up to 98% of HHT patients will have epistaxis at some point in their life. One report gives an example of a patient whose diagnosis was delayed and describes how appropriate investigations were completed, along with a brief summary of complications and available treatment options.
Surgical management of epistaxis in HHT includes conservative, medical, and surgical options. A 2011 review states that surgery remains the mainstay of treatment for refractory epistaxis in the setting of HHT, but medical management plays a vital role in prevention and treatment. A 2016 evidence-based review focuses on newer and experimental techniques for epistaxis, but does not report any specific efficacy data, survival figures, or response rates from those techniques. No controlled trial results are presented in any of these abstracts.
The abstracts are narrative reviews and single case reports. They do not provide quantitative outcomes such as reduction in epistaxis frequency, transfusion requirements, or quality-of-life measures. No drug is named or tested in these abstracts. The evidence base for any specific medical or surgical intervention in HHT epistaxis remains limited to expert opinion and descriptive case series.
What is still missing are randomised controlled trials with standardised outcome measures, adequate sample sizes to account for the variable severity of HHT, and funding to move beyond case reports and expert algorithms. Patient stratification by genotype and severity of epistaxis is also lacking.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Dalhousie Medical Journal · 2016 · 1 citations · open access
Hereditary hemorrhagic telangiectasia: an under-recognized but potentially serious condition
AbstractHereditary hemorrhagic telangiectasia (HHT) is a vascular condition that is inherited in an autosomal dominant fashion and demonstrates variable epidemiology by region. Delays in diagnosis are common, due to the low frequency of HHT, which may lead to serious complications and poor health outcomes. The Curaçao criteria should be used to assist with the diagnosis of HHT and to identify patients who require further investigations. This report provides an example of a patient whose diagnosis of HHT was delayed, and how the appropriate investigations were completed. We also give a brief summary of the complications of HHT and available treatments options.
Epistaxis in hereditary hemorrhagic telangiectasia: an evidence based review of surgical management
AbstractAbstract Patients with Hereditary Hemorrhagic Telangiectasia (HHT) frequently present with epistaxis. Up to 98Â % of these patients will have epistaxis at some point in their life. There are multiple ways to deal with this problem, including conservative, medical and surgical options. We present a case and an update on the treatment options for HHT, with a focus on the newer and experimental techniques.
Epistaxis in hereditary hemorrhagic telangiectasia: an evidence based review of surgical management
AbstractAbstract Patients with Hereditary Hemorrhagic Telangiectasia (HHT) frequently present with epistaxis. Up to 98Â % of these patients will have epistaxis at some point in their life. There are multiple ways to deal with this problem, including conservative, medical and surgical options. We present a case and an update on the treatment options for HHT, with a focus on the newer and experimental techniques.
Life Threatening Epistaxis in Hereditary Hemorrhagic Telangiectasia: Surgical and Medical Management
AbstractObjective Provide a comprehensive review of the pathophysiology, clinical presentation, surgical treatment, and advanced medical treatment of hereditary hemorrhagic telangiectasia. Method Case study of a patient with hereditary hemorrhagic telangiectasia detailing the clinical presentation and treatment algorithm of the disease. Comprehensive literature review detailing the pathophysiology and clinical presentation, with special attention to the surgical and advanced medical management of the disease. Results A comprehensive review of hereditary hemorrhagic telangiectasia with special attention to the most up to date surgical and medical treatment regimens and algorithms. Conclusion Hereditary hemorrhagic telangiectasia creates a backdrop for life threatening epistaxis. While surgery remains the mainstay of treatment for refractory epistaxis in the setting of HHT, medical management plays a vital role in the prevention and treatment of this disease entity.
Laryngo-Rhino-Otologie · 2019 · 0 citations · open access
Nationwide call for screening by dentists for hereditary hemorrhagic telangiectasia – first results
AbstractHereditary hemorrhagic telangiectasia (HHT) is a rare inherited disease. Nosebleeds and mucocutaneous telangiectases are frequent manifestations. One of the predilection sites of the latter are oral mucosa, face and lips. The first manifestation is often delayed for decades. This is problematic as an early screening for visceral manifestations is recommended to avoid serious complications.
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.
