DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for platelet-type von Willebrand disease — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePlatelet-type von Willebrand disease maps to a 2-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 platelet-type von willebrand disease 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
cholinergic receptor nicotinic epsilon subunit (CHRNE) — CHRNE 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 clrdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9DMS · 1.92 Å · ligand CHOLESTEROL (CLR). Experimental structure, not a prediction.
What the evidence adds up to
In a 1985 report, four members of one family were identified with a new variant of type IIB von Willebrand disease, characterised by chronic thrombocytopenia, circulating platelet aggregates in vivo, and spontaneous platelet aggregation in vitro. Despite prolonged bleeding times and persistent low platelet counts, their bleeding tendency was only moderate. The underlying mechanism was described as an alteration of the von Willebrand factor molecule that increases its reactivity with platelets, leading to the removal of large von Willebrand factor multimers from plasma.
A 2020 review notes that platelet-type von Willebrand disease is a rare autosomal dominant bleeding disorder caused by a mutation in the gene for platelet glycoprotein Ib alpha, which results in enhanced binding affinity for von Willebrand factor. This abnormal binding impairs haemostatic function by clearing von Willebrand factor multimers from the circulation. Patients typically have mild thrombocytopenia with large platelets, and platelet aggregates are often visible on blood smears. The review states the condition is frequently misdiagnosed as type 2B von Willebrand disease because the two disorders are similar.
A 1978 study examined platelet function in four normal volunteers, one patient with mild von Willebrand disease, and one with a thrombocytopathy, all taking propranolol. No effect on platelet function attributable to the drug could be demonstrated in any of these subjects. The authors suggested that propranolol given in conventional doses does not impair platelet haemostatic function.
A 2025 case report describes a patient with von Willebrand disease type 2M who developed severe thrombocytopenia after surgery. The timeline suggested a direct relationship between worsening platelet count and administration of a combined factor VIII/von Willebrand factor concentrate (wilate). The report highlights the difficulty of balancing the risk of haemorrhage against the risk of thrombosis when using such concentrates in the post-surgical setting. What remains missing are prospective studies that define safe dosing thresholds for von Willebrand factor concentrates in patients with platelet-type disease, validated diagnostic criteria to distinguish it reliably from type 2B von Willebrand disease, and any trial of a drug that specifically targets the abnormal platelet-von Willebrand factor interaction.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 1985 · 79 citations · open access
Type IIB Tampa: a variant of von Willebrand disease with chronic thrombocytopenia, circulating platelet aggregates, and spontaneous platelet aggregation
AbstractType IIB von Willebrand disease is characterized by enhanced ristocetin-induced platelet aggregation and absence of large von Willebrand factor multimers from plasma. An alteration of the von Willebrand factor molecule resulting in increased reactivity with platelets appears to be the basis for these abnormalities. We have now identified a new variant of type IIB von Willebrand disease in a family in which the four affected members also have chronic thrombocytopenia, in vivo platelet aggregate formation, and spontaneous platelet aggregation in vitro. In spite of repeatedly prolonged bleeding times and persistent thrombocytopenia, their bleeding diathesis is only moderate.
American Journal of Hematology · 1978 · 14 citations
Some observations on propranolol on platelet aggregation and release
AbstractPlatelet function was investigated in four normal volunteers, one patient with a mild form of von Willebrand disease, and one with a thrombocytopathy, all taking propranolol. No effect on platelet function attributable to this drug could be demonstrated in any of these subjects. It is suggested that propranolol administered in conventional doses does not impair platelet hemostatic function.
AbstractThe results of a study of 13 families with von Willebrand’s disease, living in a small zone in the province of Vicenza are reported. 21 patients presented all diagnostic signs, while 25 showed only some of them. The aspirin tolerance test gave positive results in 7 out of 10 patients with initially normal bleeding time. Platelet aggregation studies, intended to establish the ‘threshold concentration’ of ADP and adrenaline, showed an impairment of adrenaline aggregation in 5 out of 17 patients.
Severe Thrombocytopenia in the Post-surgical Context and Administration of Factor VIIII (FVIII)/von Willebrand Factor (VWF) Concentrate in a Patient With von Willebrand Disease Type 2M
AbstractVon Willebrand disease (VWD) is the most common inherited bleeding disorder. It can be associated with a life-threatening risk of excessive bleeding in surgical procedures, and may require prophylactic treatment with a combined factor VIIII (FVIII)/von Willebrand factor (VWF) concentrate. Management of these patients may be challenging when trying to achieve the balance between avoiding the risk of haemorrhage and causing a risk of thrombosis with the treatment. We present a complex case of severe thrombocytopenia in a post-surgical setting, in which the timeline suggests a direct relationship between the worsening of platelet count and wilate administrations.
Type IIB Tampa: a variant of von Willebrand disease with chronic thrombocytopenia, circulating platelet aggregates, and spontaneous platelet aggregation
AbstractType IIB von Willebrand disease is characterized by enhanced ristocetin- induced platelet aggregation and absence of large von Willebrand factor multimers from plasma. An alteration of the von Willebrand factor molecule resulting in increased reactivity with platelets appears to be the basis for these abnormalities. We have now identified a new variant of type IIB von Willebrand disease in a family in which the four affected members also have chronic thrombocytopenia, in vivo platelet aggregate formation, and spontaneous platelet aggregation in vitro. In spite of repeatedly prolonged bleeding times and persistent thrombocytopenia, their bleeding diathesis is only moderate.
AbstractA rare autosomal dominant bleeding disorder characterized by abnormally enhanced binding of von Willebrand factor (VWF) by the platelet glycoprotein Ib receptor complex.Hemostatic function is impaired due to the removal of VWF multimers from the circulation.It is due to a mutation in the gene encoding for platelet glycoprotein Ib alpha, resulting in enhanced affinity for VWF.It is often misdiagnosed as type 2B von Willebrand disease due to similarities between these two conditions.Patients present with a mild thrombocytopenia with large platelets.Platelet aggregates are often visible in blood smears.
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.
