DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hereditary angioedema — screening already-approved drugs against its 12-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHereditary angioedema maps to a 12-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
Structures already discussed alongside hereditary angioedema in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
kallikrein B1 (KLKB1) — KLKB1 is one of the genes in this disease's Open Targets module — part of the target space DeCure's repurposing candidates point at. The protein backbone is drawn as a cartoon. The structure has benzamidine bound in it, shown as sticks.
Loading structure…
helix sheet bendrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I44 · 1.36 Å · ligand BENZAMIDINE (BEN). Experimental structure, not a prediction.
What the evidence adds up to
By 2008, no drug was approved for acute attacks of hereditary angioedema in the USA, but four agents had completed or were completing Phase III trials: plasma-derived C1 inhibitor, recombinant human C1 inhibitor, the kallikrein inhibitor DX-88, and the B2 bradykinin receptor antagonist HOE-140. Each was reported effective and safe in those trials, though the review noted that differences in mechanism and delivery might affect how physicians and patients use them. A 2010 review recapped past treatments and discussed these new options as well as potential future ones. A 2021 article described a family case and stated that human C1-esterase inhibitor showed high efficacy as pathogenetic therapy, but also emphasised that hereditary angioedema remains a significant medical and social problem because of its impact on quality of life, potential mortality, and difficulties in timely diagnosis and adequate treatment.
A 2023 case report described a 34-year-old man diagnosed with hereditary angioedema at age 10, who suffered several attacks per month. Prophylaxis with antifibrinolytic agents and androgens did not improve his condition; he used plasma-derived C1-INH concentrate or icatibant for on-demand treatment of moderate and severe attacks. At age 33 he was diagnosed with multiple sclerosis and started teriflunomide 14 mg/day. Angioedema attacks disappeared 40 days after starting treatment. The authors suggested that teriflunomide could be considered for prophylaxis but stressed that its effectiveness in hereditary angioedema should be studied further, and that underlying mechanisms of the disease are not fully clear, hindering development of effective therapies.
A 2024 Colombian study described a transdisciplinary care model for 140 patients with hereditary angioedema followed for one year. The model was associated with a 76% reduction in seizure rates, a 66% reduction in hospitalisations, and an 87% reduction in emergency room visits. Pharmacological adherence increased by 19% and was complete after four months; quality of life increased significantly. The authors concluded that hereditary angioedema requires a comprehensive approach for effective care.
What is still missing is a clear understanding of the underlying mechanisms of hereditary angioedema, which the 2023 case report explicitly states hinders the development of effective therapies. The same report notes that few cost-effective long-term prophylactic options exist, and that the single case of teriflunomide remission requires further study before it can be considered a reliable option. No large randomised controlled trial has tested teriflunomide for this indication, and no data exist on which patient subgroups might benefit. The Colombian care model shows that organisational changes can reduce attacks and hospital use, but it does not identify which specific drug or combination drives the improvement, and the study was observational, not randomised. Funding for mechanistic research and for pragmatic trials that stratify patients by genotype or biomarker status remains absent.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Allergy Asthma and Clinical Immunology · 2010 · 54 citations · open access
HAE therapies: past present and future
AbstractAdvances in understanding the pathophysiology and mechanism of swelling in hereditary angioedema (HAE) has resulted in the development of multiple new drugs for the acute and prophylactic treatment of patients with HAE. This review will recap the past treatment options, review the new current treatment options, and discuss potential future treatment options for patients with HAE.
Expert Opinion on Investigational Drugs · 2008 · 20 citations
New promise and hope for treating hereditary angioedema
AbstractBACKGROUND: While there is no approved effective therapy for the treatment of acute attacks of hereditary angioedema in the USA, four different drugs are completing or have recently completed Phase III clinical trials. OBJECTIVE: To review the clinical status and future prospects of the new therapies under development for the treatment of hereditary angioedema. METHODS: A review was carried out of the literature and presentations at meetings on the efficacy and safety of plasma-derived C1 inhibitor, recombinant human C1 inhibitor, the kallikrein inhibitor DX-88, and the B2 bradykinin receptor antagonist HOE-140. RESULTS/CONCLUSION: Each of these drugs has been shown to be effective and safe for the treatment of hereditary angioedema; however, subtle differences in their mechanisms of action and delivery may influence how physicians and patients utilize the different drugs. The availability of effective therapy is expected to reshape the management of hereditary angioedema.
Successful Use of Oxandrolone in the Prophylaxis of Hereditary Angioedema: A Case Report
AbstractHereditary angioedema (HAE) is a rare autosomal dominant disorder typified by a deficiency or dysfunction of the C1-esterase inhibitor (Cl-INH), and characterized clinically by swelling of the extremities, severe episodic abdominal pain and sometimes upper airway obstruction. This paper reports for the first time the successful use of oxandrolone in the prophylaxis of HAE in a 14-year-old girl. Oxandrolone has comparatively milder side effects and less potential for hepatotoxicity and virilization than other attenuated androgens used in the prophylaxis of this disease. We believe oxandrolone should be considered as an alternative androgen therapy for children and adults with HAE, particularly females experiencing untoward side effects from danazol or stanozolol, and patients who are not adequately controlled on maximum doses of androgens currently prescribed for HAE.
Allergy and Asthma Proceedings · 2011 · 8 citations
Recurrent attacks of hereditary angioedema: A case of delayed diagnosis
AbstractHereditary angioedema is a rare disorder, and patients frequently endure long duration of symptoms, frequent physician visits, and unnecessary procedures prior to a diagnosis. Patients with novel mutations may experience especially long delays in diagnosis due to a lack of family history. This case demonstrates one such case in which diagnosis was delayed for many years. Improved physician awareness of the signs and symptoms of hereditary angioedema may prevent such delay for patients with this disorder in the future. Abdominal pain, angioedema, bradykinin, C1 inhibitor, hereditary, inherited, swelling.
Hereditary Angioedema : A Literature Review and National Management Guidelines
AbstractBACKGROUND: Hereditary angioedema, a rare and potentially life-threatening condition, is the result of a defect in the C1 esterase inhibitor. Primary care physicians should be familiar with this condition to avoid complications and improve quality of care. METHODS: We present two cases of hereditary angioedema followed by a discussion based on a literature review of the recent guidelines and advances in this condition. OBJECTIVES: To highlight the clinical aspects, diagnosis and treatment of this condition and propose a practical local management based on the available medication. CONCLUSION: Hereditary angioedema management is still evolving. More efforts should be made concerning the drug therapy which is very costly and not available worldwide.
[Design and implementation of a transdisciplinary care model for patients with hereditary angioedema, in a Colombian health institution].
AbstractOBJECTIVE: Describe the design and implementation of a transdisciplinary care model for patients with hereditary angioedema in Colombia. METHODS: Descriptive longitudinal observational study. 140 patients with hereditary angioedema were included in a transdisciplinary care model for one year. Seizure rates, hospitalizations, emergency room visits, quality of life, and pharmacological adherence were measured. RESULTS: The model was associated with reductions of 76% in seizures, 66% in hospitalizations, and 87% in emergency room visits. Pharmacological adherence increased 19% and was complete after four months. The quality of life increased significantly. CONCLUSIONS: Hereditary angioedema is an orphan disease that requires a comprehensive approach for effective care.
Meditsinskiy sovet = Medical Council · 2021 · 0 citations · open access
Hereditary angioedema: approaches to diagnosis and treatment, analysis of a clinical family case
AbstractHereditary angioedema belongs to the group of rare, orphan, genetically determined defects that represent a significant medical and social problem due to the pronounced impact on the quality of life and potential mortality, as well as the emerging difficulties associated with timely diagnosis and the appointment of adequate treatment. The article presents data on the modern classification of hereditary angioedema, clinical manifestations of the disease, approaches to diagnosis verification and treatment principles. Therapy of hereditary angioedema is determined by the need for effective relief of acute attacks of the disease, prevention of edema before medical interventions, and, if indicated, long-term prophylaxis. The article discusses a differentiated approach to the treatment of hereditary angioedema, characterizes various options for therapeutic interventions. In a clinical case, the history of several generations of a family with manifestations of hereditary angioedema is described. A modern approach to the diagnosis of the disease based on detailed history, clinical symptoms, and laboratory research results has been demonstrated. The analysis of the effectiveness of treatment was carried out and the high efficiency of pathogenetic therapy of hereditary angioedema with human C1-esterase inhibitor was shown.
European Journal of Case Reports in Internal Medicine · 2023 · 0 citations · open access
Remission of hereditary angioedema attacks associated with starting teriflunomide in a patient with multiple sclerosis
AbstractBackground: Hereditary angioedema is a rare hereditary and potentially life-threatening disorder characterized by recurrent attacks of cutaneous and submucosal swelling. In spite of the advances made in terms of pathophysiology, underlying mechanisms are not fully clear and this, in turn, hinders the development of effective therapies. Currently, on demand treatment is considered first-class, with few cost-effective, long-term prophylactic options. Case presentation: Here we describe the case of a 34-year-old man diagnosed with hereditary angioedema at the age of 10, who used to suffer several angioedema attacks per month. He was given prophylactic treatment with antifibrinolytic agents and androgens without improvement. Moreover, he was treated with plasma-derived C1-INH concentrate or icatibant for on-demand treatment of moderate and severe angioedema attacks. At the age of 33, after suffering sudden vision loss and lower limb paresthesia, he was studied and diagnosed with multiple sclerosis. Teriflunomide was administered at a dosage of 14 mg/day. Angioedema attacks disappeared 40 days after starting treatment. Conclusion: Thus, we suggest considering the pathophysiologic mechanisms on which teriflunomide could be active and consider this drug carefully as an option for prophylaxis purposes. Yet, its effectiveness on this condition should be further studied. LEARNING POINTS: Underlying mechanisms in hereditary angioedema lack clarity and hence hinder the development of effective therapies.On-demand treatment of hereditary angioedema is considered first class, with few cost-effective, long-term prophylactic options.The mechanisms of action and effectiveness of teriflunomide on hereditary angioedema should be studied further.
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.
