DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for aplastic anemia — screening already-approved drugs against its 20-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAplastic anemia maps to a 20-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 aplastic anemia 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
hemoglobin subunit beta (HBB) — HBB 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 hemdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 1DXT · 1.7 Å · ligand PROTOPORPHYRIN IX CONTAINING FE (HEM). Experimental structure, not a prediction.
What the evidence adds up to
In 1968, four consecutive children with acquired aplastic anaemia, including one who had not responded to prolonged testosterone treatment, all responded to the synthetic anabolic drug oxymetholone. Three of the four stopped all medication and remained well for eighteen to twenty-four months. A fifth child with constitutional aplastic anaemia who had also failed testosterone therapy improved on oxymetholone. The only reported side effect was mild virilisation. Significant improvement in peripheral blood counts was noted in all five children during the second and third month of treatment. The authors described oxymetholone as a potent bone marrow stimulant.
A 2024 study of 16 patients with aplastic anaemia compared the results of immunosuppressive therapy and splenectomy. The authors reported that the treatment outcomes in the two groups did not have statistically significant differences. They concluded that there remain enough problems in this field to require further study.
A 2000 review noted that improvements in bone marrow transplantation and immunosuppression had increased the number of long-term survivors, shifting concern from immediate survival to late malignant and non-malignant complications. A 2019 review described acquired aplastic anaemia as an immune-mediated bone marrow failure syndrome and discussed the abnormal immunologic mechanisms thought to mediate its pathogenesis, aiming to provide a theoretical basis for clinical treatment.
What is still missing are large, controlled trials that can determine whether any single intervention—oxymetholone, splenectomy, or specific immunosuppressive regimens—offers a clear advantage over others in defined patient subgroups. The 1968 oxymetholone series had only five children and no control group. The 2024 splenectomy study was too small to detect a statistically significant difference, and the authors themselves said more work is needed. No modern trial has yet stratified patients by immune profile or genetic subtype to test whether the immune mechanisms described in the 2019 review can guide treatment selection.
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 · 1968 · 64 citations · open access
Oxymetholone Therapy in Aplastic Anemia
AbstractAbstract Four consecutive children with acquired aplastic anemia, including one refractory to prolonged testosterone treatment, have responded to the synthetic anabolic drug oxymetholone. Three of these four are now off all medication and have remained well for eighteen to twenty-four months. A fifth child, with constitutional aplastic anemia refractory to testosterone therapy, has also improved while on oxymetholone. Mild virilization was the only side effect noted. Significant improvement in peripheral blood was noted in all five children during the second and third month of treatment. Oxymetholone appears to be a potent bone marrow stimulant.
Cure from Severe Aplastic Anemia in vivo and Late Effects
AbstractRecent progress in the treatment of aplastic anemia has dramatically changed the previously grim prognosis for these patients. Improvements in bone marrow transplantation and immunosuppression have increased the number of long-term survivors so that immediate survival is no longer the sole concern. Here, were review the major clinical studies and summarize recent analyses of late malignant and nonmalignant complications following treatment of aplastic anemia.
International Journal for Research in Applied Science and Engineering Technology · 2024 · 0 citations · open access
Efficacy of Splenectomy for Aplastic Anemia
AbstractAbstract: The article presents the results of a study of 16 patients with aplastic anemia who were treated in the hematology department of the Republican Clinical Hospital for 3 years. The efficacy of immunosuppressive therapy and splenectomy in this category of patients was assessed by statistical analysis of laboratory parameters at admission and discharge. During the study, data were obtained showing that the results of treatment in the two groups of patients did not have statistically significant differences, therefore, it was determined that there are enough problems to be studied in this field in the future, and solutions to some of these problems were given.
Guoji shuxue ji xueyexue zazhi · 2019 · 0 citations
Research progress of abnormal immunologic mechanism in acquired aplastic anemia
AbstractAplastic anemia (AA) is considered as an immune-mediated bone marrow failure syndrome, characterized by peripheral pancytopenia and destruction of hematopoietic stem/progenitor cells. Acquired aplastic anemia (aAA) is more common in clinic. Abnormal immunity is one of the major factors mediating the pathogenesis of aAA. This review discusses the current understanding of the immunobiology in aAA, so as to clarify the immune mechanism of this disease, and to provide a theoretical basis for the clinical treatment of aAA.
Key words:
Anemia, aplastic; Immunity; Immunosuppressive agent; Children; Acquired aplastic anemia
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.
