DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for gray platelet syndrome — 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 moduleGray platelet syndrome 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 gray platelet syndrome 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
Gray platelet syndrome is a rare inherited disorder characterised by a marked decrease or absence of alpha-granules and platelet-specific alpha-granule proteins, as shown in a 1985 study of two patients. Those patients’ platelets responded to alpha-thrombin with a normal, dose-dependent membrane potential change and a normal secondary release of a marker thought to be released from lysosomal granules, indicating that the initial thrombin response does not require alpha-granules or release of their contents. A 2019 case report notes that bleeding intensity in gray platelet syndrome does not correlate with platelet count nor with functional test results, and describes perioperative bleeding management in a patient requiring multiple redo cardiac surgeries.
The 2025 review on thrombocytopenia defines it as a platelet count below 150,000/μL and lists causes including pregnancy (7–10%), immune-mediated diseases, certain drugs, infections, and systemic and haematologic diseases. It states that treatment depends on the cause and severity, and can include discontinuing precipitating drugs, immunosuppressive drugs, or thrombopoietin receptor agonists in immune-mediated cases. Prophylactic platelet transfusion can be considered if the count drops below 10,000–20,000/μL, or below 50,000/μL before an intervention or operation, but not in immune thrombocytopenic purpura or thrombotic thrombocytopenic purpura.
A 2013 case report describes probable carvedilol-induced thrombocytopenia in a 64-year-old woman whose platelet count dropped to 94,000/mm³ after carvedilol was added to her regimen. After carvedilol was discontinued and replaced with metoprolol, her platelet count rose to 319,000/mm³ at discharge and remained stable. The recovery time coincided with carvedilol’s elimination half-life. A 2018 retrospective study of 471 preterm infants found that lower platelet counts during pharmacological therapy for patent ductus arteriosus were independently associated with treatment failure, but platelet counts before initiation of therapy did not affect outcome, and no specific platelet cutoff value could predict treatment failure.
What is still missing for gray platelet syndrome specifically: no controlled trials of any drug for the condition exist; management relies on case reports and expert opinion; the 2019 case report describes perioperative care but not a systematic treatment protocol; patient stratification by bleeding phenotype or genotype is not established; and funding for rare platelet disorder trials remains scarce.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Frontiers in Pediatrics · 2018 · 19 citations · open access
Association between Platelet Counts before and during Pharmacological Therapy for Patent Ductus Arteriosus and Treatment Failure in Preterm Infants
AbstractBACKGROUND: The role of platelets for mediating closure of the ductus arteriosus in human preterm infants is controversial. Especially, the effect of low platelet counts on pharmacological treatment failure is still unclear. METHODS: In this retrospective study of 471 preterm infants [<1,500 g birth weight (BW)], who were treated for a patent ductus arteriosus (PDA) with indomethacin or ibuprofen, we investigated whether platelet counts before or during pharmacological treatment had an impact on the successful closure of a hemodynamically significant PDA. The effects of other factors, such as sepsis, preeclampsia, gestational age, BW, and gender, were also evaluated. RESULTS: < 0.05). Receiver operating characteristic (ROC) curve analysis showed that platelet counts after the first, and before and after the second cyclooxygenase inhibitor (COXI) cycle were significantly associated with treatment failure (area under the curve of >0.6). However, ROC curve analysis did not reveal a specific platelet cutoff-value that could predict PDA treatment failure. Multivariate logistic regression analysis showed that lower platelet counts, a lower BW, and preeclampsia were independently associated with COXI treatment failure. CONCLUSION: We provide further evidence for an association between low platelet counts during pharmacological therapy for symptomatic PDA and treatment failure, while platelet counts before initiation of therapy did not affect treatment outcome.
American Journal of Health-System Pharmacy · 2013 · 5 citations
Probable carvedilol-induced thrombocytopenia
AbstractPURPOSE: A case of probable carvedilol-induced thrombocytopenia is reported. SUMMARY: A 64-year-old African-American woman with a history of hypertension, diastolic dysfunction, mild left-ventricular hypertrophy, and pulmonary embolism was hospitalized with a platelet count of 94,000 platelets/mm(3). Dalteparin, warfarin, and carvedilol had recently been added to her medication regimen. Beta-2-glycoprotein immunoglobulin G (IgG) antibody, anticardiolipin IgG, and anticardiolipin immunoglobulin M tests, conducted to rule out antiphospholipid syndrome, revealed values within the normal range. After the exclusion of dalteparin, hydrochlorothiazide, and other causes of drug- and non-drug-related thrombocytopenia, carvedilol was discontinued and replaced with metoprolol tartrate. After this substitution, the patient's platelet count continued to rise. On hospital day 10, the patient was discharged to home on low-molecular-weight heparin bridging therapy, warfarin sodium 5 mg orally daily, ranitidine 150 mg orally daily, metoprolol tartrate 75 mg orally twice daily, lisinopril 20 mg orally daily, amlodipine 10 mg orally daily, cyanocobalamin 1000 mg orally daily, and two tablets of hydrochlorothiazide 25 mg-triamterene 37.5 mg orally daily. Her platelet count was 319,000 platelets/mm(3) on the day of discharge and remained stable thereafter. The recovery time of the platelets coincided with the elimination half-life of carvedilol. CONCLUSION: A woman developed thrombocytopenia, first noticed as a reduction in the platelet count to a low-normal value, 17 days after treatment with carvedilol was begun. Other possible culprit drugs were withdrawn, but the platelet count continued to drop until carvedilol was discontinued. The platelet count rose on the day of carvedilol removal and was within the normal range within another day.
British Journal of Haematology · 1985 · 5 citations
Studies of platelets from patients with the grey platelet syndrome
AbstractThe grey platelet syndrome is a rare inherited disorder characterized by a marked decrease or absence of alpha-granules and of platelet-specific alpha-granule proteins. By utilizing platelets from two patients with this syndrome, we here demonstrate that the initial response of human platelets to alpha-thrombin does not require the presence of alpha-granules nor the effective release of their constituents. Furthermore, these platelets respond to thrombin with a normal, dose-dependent membrane potential change, and a normal secondary release of diS-C3-(5) thought to be released in parallel with beta-glucuronidase from the lysosomal granules. These results give new insight into the initial steps in the thrombin response of normal platelets.
Gray platelet syndrome: Management of perioperative bleeding in redo cardiac surgery
AbstractGray platelet syndrome (GPS) is a rare (<1/1 000 000) and inherited platelet function disorder characterized by macrothrombocytopenia, α-granule deficiency, and hemorrhages. Bleeding intensity does not correlate with platelet count nor with functional test results. We hereby describe the perioperative bleeding prevention and management of a patient with GPS requiring multiple redo cardiac surgeries.
Deutsches Ärzteblatt international · 2025 · 1 citations
The differential diagnosis of thrombocytopenia
AbstractBACKGROUND: Thrombocytopenia is defined as a platelet count below 150 000/μL. It increases the risk of bleeding, often due to an existing underlying condition. A meticulous diagnostic evaluation is needed so that specific treatment can be initiated and complications avoided. METHODS: This review is based on clinical studies up to June 2025 that were retrieved by a selective search with pertinent key words in the MEDLINE/PubMed database, and on current guidelines. RESULTS: Thrombocytopenia often arises in association with pregnancy (7-10 %), immune-mediated diseases such as idiopathic thrombocytopenic purpura (ITP), certain drugs (e.g., heparin-induced thrombocytopenia [HIT] in as many as 1% of patients treated with unfractionated heparin), infections, and systemic and hematologic diseases. The diagnostic evaluation is by an algorithm involving the clinical history, a complete blood count, and other, specific tests. Emergencies such as thrombotic microangiopathy (TMA) and disseminated intravascular coagulation require rapid therapeutic intervention. The treatment depends on the cause and severity of thrombocytopenia; it can include the discontinuation of precipitating drugs, the use of immunosuppressive drugs or thrombopoietin receptor agonists in immune-mediated cases, and specific measures against infection, TMA, or malignant diseases. Prophylactic platelet transfusion can be considered if the platelet count drops below 10 000-20 000/μL, or below 50 000/μL before an intervention or operation, but not in cases of ITP or thrombotic thrombocytopenic purpura (TTP). CONCLUSION: The treatment depends on the clinical manifestations, platelet count, and underlying cause.
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.
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