Rare & Orphan Lab · DeCure for X

DeCure for Hematologic disease

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hematologic disease — screening already-approved drugs against its 38-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module38 genesLead labRare & Orphan
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The disease map

Disease moduleHematologic disease maps to a 38-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 hematologic 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

ring finger protein 4 (RNF4)RNF4 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 frudrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 2XEU · 1.5 Å · ligand beta-D-fructofuranose (FRU). Experimental structure, not a prediction.

What the evidence adds up to

The abstracts provided are largely textbook reviews, book notices, and a technology-development poster, not clinical trial reports. The 1988 editions of Blood: A Textbook of Hematology and Molecular Basis of Blood Diseases describe the field's state at that time, with the latter noting that molecular cloning and gene-structure analysis had generated information "unavailable, and, in some instances, unimaginable, a decade ago." A 2018 volume reiterates that haematology advances in parallel with technology, allowing "more effective and less toxic alternative therapeutic approaches directed against critical molecular pathways," but gives no specific drug, dose, or outcome data. A 1997 review mentions the "increasingly important role of hematopoietic growth factors" in supportive care, again without quantitative results.

A 2022 case series of 11 COVID-19 patients with retroperitoneal haematoma and subcutaneous ecchymosis reports that increased INR and bleeding required discontinuation of anticoagulants, with fresh frozen plasma and "essential and tri amino injections" given; no survival or recovery rates are stated. The two identical 2022 abstracts on APTASHAPE describe a blood-profiling technology using RNA aptamers to generate protein and metabolite imprints from 1 microlitre of plasma, applied to non-alcoholic fatty liver disease and SARS-CoV-2 severity prediction; no sensitivity, specificity, or patient numbers are given, and the work is not specific to haematologic malignancy.

No abstract reports a drug repurposed for haematologic disease with measurable efficacy. The only interventional data concern anticoagulant cessation in COVID-19 coagulopathy, and that is a management caution, not a treatment recommendation. What is missing is any controlled trial, any patient stratification by molecular subtype, and any funding commitment to test the aptamer platform or growth-factor regimens against haematologic endpoints. Without those, the literature here offers no basis for repurposing a specific agent.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Annals of Internal Medicine · 1988 · 199 citations

Blood: A Textbook of Hematology.

AbstractThis edition of the benchmark text is more a complete revitalization than a revision: over 75 per cent of the text and 80 per cent of the references are new. Clearly written and abundantly illustrated, it presents the pathogenesis, clinical manifestations, and management of all hematologic disorders. It follows a logical organization from definition to treatment, and descriptions are written in language common to both clinical and basic science, making it as accessible to the general internist as to the specialist

https://doi.org/10.7326/0003-4819-108-3-504_5
Annals of Internal Medicine · 1988 · 12 citations · open access

Molecular Basis of Blood Diseases.

AbstractThe recent application of the techniques of molecular biol- ogy to the study of human diseases has made it possible to obtain information unavailable-and, in some instances, unimaginable-a decade ago.Because of the rapidity with which developments have occurred, many clinicians and scientists may be unaware of them and of the insights they provide into the origin of diseases.To address the needs of this audience, four noted authorities in the field, George Stamatoyannopoulos, Arthur Nienhuis, Philip Leder, and Philip Majerus, have recruited 27 of their colleagues to describe what is currently known about the molecular basis of blood diseases.The first chapter introduces the reader to the methods of molecular cloning and analysis of gene structure that have generated much of the information pre- sented subsequently.Several chapters then describe gene structure, expression, and regulation, using the globin genes as a model.After this general introduction, specific groups of diseases are addressed.Each area of classic hematology-the red cell, the white cell (both normal and malignant), and the soluble and cellular components of the hemostatic system (including hemostasis, thrombosis, and fibrinolysis)-is taken up in turn.In addition, three chap- ters present immunological topics-immunoglobulin and T-cell receptor genes (as well as lymphocyte differentia- tion), enzyme deficiencies associated with immunological disorders, and molecular mechanisms of complement ac-

https://doi.org/10.7326/0003-4819-108-3-504_8
InTech eBooks · 2018 · 4 citations

Hematology - Latest Research and Clinical Advances

AbstractHematology has constantly been advancing in parallel with technological developments that have expanded our understanding of the phenotypic, genetic, and molecular complexity and extreme clinical and biological heterogeneity of blood diseases. This has in turn allowed for developing more effective and less toxic alternative therapeutic approaches directed against critical molecular pathways. The continuous and rather extensive influx of new information regarding the key features and underlying mechanisms as well as treatment options in hematology requires a frequent update of this topic. The primary objective of this book is to provide the specialists involved in the clinical management and experimental research in hematological diseases with comprehensive and concise information on some important theoretical and practical developments in the biology, clinical assessment, and treatment of patients, as well as on some molecular and pathogenetic mechanisms and the respective translation into novel therapies.

https://doi.org/10.5772/intechopen.70388
Current Opinion in Hematology · 1997 · 2 citations

Supportive care in hematologic malignancies, including hematopoietic growth factors, transfusions, and infectious complications

AbstractThis review focuses on some of the recent developments in the field of supportive care in hematologic malignancies. The increasingly important role of hematopoietic growth factors in modern treatment modalities for hematologic disorders is clear. Newer developments in transfusional therapy and in the management of infectious complications are also discussed.

https://doi.org/10.1097/00062752-199704040-00005
Annals of Medicine and Surgery · 2022 · 0 citations · open access

Acute Progressive retro-peritoneal hematoma in COVID19 patients with sub cutaneous ecchymosis

AbstractIntroduction: and importance: COVID19 is a multifunction disease where hematological disorders are reported. Coagulopathy is seen in these patients, indicated by thromboembolic events. Case presentation: We present case of 11 COVID19 who were presented with localized skin ecchymosis lesions in different areas of the body and retroperitoneal hematoma in the posterior wall of the abdomen and chest. Clinical discussion: Increased INR and bleeding and ecchymosis in Corona patients require discontinuation of anticoagulants and, FFP, essential and tri amino injections are needed. Conclusions: Diagnosis and management of hematoma is important to avoid fatality.

https://doi.org/10.1016/j.amsu.2022.104107
British Medical Bulletin · 1964 · 0 citations

Book Reviews

AbstractBook Reviews Get access Diagnosis and Treatment of Blood Diseases M. C. G. Israëls. London: Helnemann Medical Books, 1963. vii+204 pages; II plates. 22×15 cm.£1 15s. J. L. Stafford J. L. Stafford Search for other works by this author on: Oxford Academic PubMed Google Scholar British Medical Bulletin, Volume 20, Issue 3, September 1964, Page 250, https://doi.org/10.1093/oxfordjournals.bmb.a070346 Published: 01 September 1964

https://doi.org/10.1093/oxfordjournals.bmb.a070346
Zenodo (CERN European Organization for Nuclear Research) · 2022 · 0 citations · open access

Aptamer-based blood profiling for diagnosis, treatment evaluation and disease severity prediction

AbstractThe poster was presented at The personalised medicine network, Second Annual Meeting, 8th November 2022. Abstract: Daniel Miotto Dupont1, Søren Fjelstrup1, <strong>Asger Jørgensen1</strong>, Claus Bus1, Mikkel Kjær4, Henning Grønbæk4, Christian Vægter3, Nadia Goncalves3, Thomas Benfield5, Peter Garred6, Maria Møller5, Simone Israelsen5, Håkon Sandholdt5, Cecilie Hansen6, Rasmus Thomsen7, Natasha Michaelsen7, Peter Heegaard8 and Jørgen Kjems1,2. <br> 1Interdisciplinary Nanoscience Center (iNANO), 2Department of Molecular Biology and Genetics, 3Department of Bio-medicine, Aarhus University, 4Department of Clinical Medicine, Aarhus University Hospital, 5Department of Infectious Diseases, Amager and Hvidovre Hospital, 6Rigshospitalet Copenhagen, 7Diabetes and Cardiovascular Research, Novo Nordisk, 8DTU Health Tech The molecular composition of blood is a reflection of our physiology, health state, lifestyle etc., in many ways a highway of biological information difficult to access. We developed a novel technology, APTASHAPE, to translate this "difficult-to-read" language into readable digital information. APTASHAPE involves the use of panels of small biosensor molecules, 2’-fluoro protected RNA aptamers, to generate imprints of the protein and metabolite signature in a plasma sample. We applied APTASHAPE to profile different cohorts of samples from patients and animal models, and here we present our results in relation to diagnosis and treatment evaluation within Non-Alcoholic Fatty Liver Disease (NAFLD) as well as prediction of clinical disease severity for patients hospitalised following SARS CoV-2 infection. The technology possesses the potential to measure several disease conditions simultaneously based on a single drop of blood (1 microliter of plasma) and can serve as a health-state measuring and prediction tool. The APTASHAPE technology allows individual evalua-tion of patients and tailors personalised medicine based on the molecular composition of the patient's blood. *The poster is a modified version of the poster previously presented (https://doi.org/10.5281/zenodo.7568262). Contact information: Asger Givskov Jørgensen<br> [email protected]<br>

https://doi.org/10.5281/zenodo.7573218
Zenodo (CERN European Organization for Nuclear Research) · 2022 · 0 citations · open access

Aptamer-based blood profiling for diagnosis, treatment evaluation and disease severity prediction

AbstractThe poster was presented at The personalised medicine network, Second Annual Meeting, 8th November 2022. Abstract: Daniel Miotto Dupont1, Søren Fjelstrup1, <strong>Asger Jørgensen1</strong>, Claus Bus1, Mikkel Kjær4, Henning Grønbæk4, Christian Vægter3, Nadia Goncalves3, Thomas Benfield5, Peter Garred6, Maria Møller5, Simone Israelsen5, Håkon Sandholdt5, Cecilie Hansen6, Rasmus Thomsen7, Natasha Michaelsen7, Peter Heegaard8 and Jørgen Kjems1,2. <br> 1Interdisciplinary Nanoscience Center (iNANO), 2Department of Molecular Biology and Genetics, 3Department of Bio-medicine, Aarhus University, 4Department of Clinical Medicine, Aarhus University Hospital, 5Department of Infectious Diseases, Amager and Hvidovre Hospital, 6Rigshospitalet Copenhagen, 7Diabetes and Cardiovascular Research, Novo Nordisk, 8DTU Health Tech The molecular composition of blood is a reflection of our physiology, health state, lifestyle etc., in many ways a highway of biological information difficult to access. We developed a novel technology, APTASHAPE, to translate this "difficult-to-read" language into readable digital information. APTASHAPE involves the use of panels of small biosensor molecules, 2’-fluoro protected RNA aptamers, to generate imprints of the protein and metabolite signature in a plasma sample. We applied APTASHAPE to profile different cohorts of samples from patients and animal models, and here we present our results in relation to diagnosis and treatment evaluation within Non-Alcoholic Fatty Liver Disease (NAFLD) as well as prediction of clinical disease severity for patients hospitalised following SARS CoV-2 infection. The technology possesses the potential to measure several disease conditions simultaneously based on a single drop of blood (1 microliter of plasma) and can serve as a health-state measuring and prediction tool. The APTASHAPE technology allows individual evalua-tion of patients and tailors personalised medicine based on the molecular composition of the patient's blood. *The poster is a modified version of the poster previously presented (https://doi.org/10.5281/zenodo.7568262). Contact information: Asger Givskov Jørgensen<br> [email protected]<br>

https://doi.org/10.5281/zenodo.7573217

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works using Disease Ontology synonyms, 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.