DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for von Willebrand disease 3 — 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 moduleVon Willebrand disease 3 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 von willebrand disease 3 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.
What the evidence adds up to
Von Willebrand disease type 3 is the most severe form of the most common inherited coagulation disorder, caused by mutations on chromosome 12 in the region p13.2 that encodes von Willebrand factor. The molecular biology of the VWF gene has been characterised, but as of 2015 these techniques had not been integrated into routine diagnostic testing, and even in tertiary centres in Mexico patients did not have a definitive diagnosis. Analysis of VWF multimers meets diagnostic requirements but is not easy to standardise. The disease is highly heterogeneous because different molecular mechanisms produce different clinical and laboratory phenotypes.
Two paediatric patients with type 3 disease who needed surgery at a Russian clinic between 2018 and 2019 received Haemate P as short-term prophylaxis. The application was reported as 100% successful in both cases. No larger series or controlled data were provided.
A phase 3, open-label, international multicentre study was registered to investigate efficacy and safety of prophylactic treatment with recombinant von Willebrand factor (vonicog alfa) in adults with severe von Willebrand disease. The study was designed to assess immunogenicity, thrombogenicity, hypersensitivity reactions, pharmacokinetics, health-related quality of life, and pharmacoeconomics. No results from this study had been published in the abstracts provided.
What is still missing are completed phase 3 trial results for recombinant VWF prophylaxis, any controlled data on surgical prophylaxis in type 3 patients beyond two case reports, standardised molecular diagnostic methods that are accessible outside specialised centres, and studies that stratify patients by the specific molecular defect causing their disease.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Cirugía y Cirujanos · 2015 · 9 citations · open access
Enfermedad de von Willebrand, biología molecular y diagnóstico
AbstractLa enfermedad de von Willebrand es el trastorno hereditario más frecuente de las proteínas de la coagulación en los seres humanos. Existen 3 tipos: 1, 2A, 2B, 2N, 2M, y 3. Está asociada a mutaciones en el cromosoma 12, en la región p13.2, que codifica para el factor de von Willebrand (VWF), el cual se sintetiza en las células endoteliales y megacariocitos. La biología molecular ha permitido la caracterización del gen del VWF, adquiriendo un papel importante en el diagnóstico la enfermedad de von Willebrand así como en la investigación de alteraciones en otros genes, que pueden estar involucrados en la regulación de la síntesis, procesamiento y secreción del VWF. Sin embargo, aún no se han integrado las estrategias de biología molecular entre las pruebas de diagnóstico disponibles. El análisis de los multímeros del VWF es una metodología que cumple con las características para el diagnóstico, pero no es fácil de estandarizar. Tomando en consideración que aún en los centros de tercer nivel en nuestro país los enfermos de von Willebrand no cuentan con un diagnóstico definitivo, es necesario implementar estas metodologías para su estudio y mejorar su diagnóstico. La enfermedad de von Willebrand es heterogénea debido a los mecanismos moleculares que producen los distintos fenotipos clínicos y de laboratorio. En México existen pocos trabajos relacionados con esta enfermedad, por ello es fundamental realizar un estudio integral que incluya aspectos clínicos, pruebas de laboratorio básicas y especiales, para establecer el diagnóstico correcto, desarrollar nuevos enfoques terapéuticos, y así ofrecer atención médica y asesoramiento genético adecuados. Von Willebrand disease is the most common inherited disorder of the coagulation proteins in humans. There are three types: 1, 2A, 2B, 2N, 2M and 3. It is associated with mutations on chromosome 12 in the region p13.2, encoding the von Willebrand factor (VWF), which is synthesized in endothelial cells and megakaryocytes. The VWF gene has been characterised using molecular biology techniques, which have acquired an important role in diagnosis von Willebrand disease, as well as in the investigation of alterations in other genes, which may be involved in regulating the synthesis, processing, and secretion of VWF. However, there are still no strategies to integrate the molecular biology diagnostic tests available. Analysis of VWF multimers is a methodology that meets the characteristics for diagnosis, but it is not easy to standardise. Considering that even in tertiary centres in our country, von Willebrand patients do not have a definitive diagnosis, it is necessary to implement these methodologies to study and improve diagnosis. Von Willebrand disease is highly heterogeneous due to the molecular mechanisms that produce the various clinical and laboratory phenotypes. In Mexico there are few studies related to this disease; therefore it is essential to conduct a comprehensive study including clinical, basic, and special testing laboratory tests, in order to establish a correct diagnosis, develop new therapeutic approaches, and offer the appropriate medical care and genetic counselling.
AbstractThe article entitled ‘Diagnosis and treatment of von Willebrand disease: new perspectives and nuances’, by C. M. Kessler, Haemophilia 2007; 13(Suppl 5): 3–14, included two tables (1 and 2), which were attributed to the National Heart Lung Blood Institute Consensus Statement for the Guidelines for the Treatment and Diagnosis of von Willebrand disease. We regret that these tables were published in advance of the publication of the NHLBI guidelines in Haemophilia 2008; 14: 171–232. The intent was to publish both the guidelines and the supplement concurrently in the same issue; however, because of editorial delays, the coordinated publication did not occur as planned. The editors and publishers apologize for this inadvertent breach of confidentiality of prepublished data and have instituted procedures intended to prevent this from recurring.
Summary of a Nordic Workshop on von Willebrand Disease
AbstractThis supplement is a summary of a Nordic Workshop on von Willebrand disease held in Malmo, Sweden in August 2004. The meeting was a follow-up of the Nordic von Willebrand Symposium, held on Aland in September 1998 and, as such, was arranged by the Nordic von Willebrand group established after this symposium. Speakers from the USA, Germany, England, Denmark, Norway and Sweden covered different aspects of von Willebrand disease (VWD), from prevalence, classification and diagnostic aspects, to treatment and management.
Russian Journal of Pediatric Hematology and Oncology · 2020 · 0 citations · open access
Successful surgical interventions in pediatric patients with type 3 disease of von Willebrand
AbstractThere were two pediatric patients under our supervision at Saint-Petersburg State Pediatric Medical University clinic from 2018 to 2019 with type 3 disease of von Willebrand, who needed the surgical treatment. The surgery was made with Haemate® P which is used as for long-term prophylaxis, as for short-term prophylaxis with surgical treatment for disease of von Willebrand. Application of Haemate® P was 100 % successful in both cases.
Available datapackage for study 'A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE'
AbstractThe purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity, thrombogenicity and hypersensitivity reactions, as well as pharmacokinetics (PK), health related quality of life (HRQoL) and pharmacoeconomics of prophylactic treatment with recombinant von Willebrand factor (rVWF) (vonicog alfa) in adult participants with severe von Willebrand disease (VWD).
A PROSPECTIVE, PHASE 3, OPEN-LABEL, INTERNATIONAL MULTICENTER STUDY ON EFFICACY AND SAFETY OF PROPHYLAXIS WITH rVWF IN SEVERE VON WILLEBRAND DISEASE
AbstractThe purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity, thrombogenicity and hypersensitivity reactions, as well as pharmacokinetics (PK), health related quality of life (HRQoL) and pharmacoeconomics of prophylactic treatment with recombinant von Willebrand factor (rVWF) (vonicog alfa) in adult participants with severe von Willebrand disease (VWD).
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.
