DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for megaloblastic anemia — screening already-approved drugs against its 15-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMegaloblastic anemia maps to a 15-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
approvedCyanocobalaminApproved drugapprovedHydroxocobalaminApproved drug
Structures already discussed alongside megaloblastic anemia in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
The surface-exposed lipo-protein of BtuG2 — Hydroxocobalamin has a real, experimentally solved structure in complex with this target (PDB 8BB0, 1.5 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet i2adrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8BB0 · 1.5 Å · ligand Hydroxocobalamin (I2A). Experimental structure, not a prediction.
What the evidence adds up to
Twenty-one children with megaloblastic anaemia, aged two months to 15 years, were studied in a one-year hospital-based analysis. The authors concluded that the condition is a common cause of undiagnosed anaemia and that treatment is simple and affordable, but they provided no numerical data on response rates, survival, or specific haematological values. They warned that untreated disease leads to morbidity from anaemia and neurological involvement.
A cross-sectional study of 100 patients with megaloblastic anaemia reported that 51% were male and 49% female. Pancytopenia occurred in 57% of patients, anaemia alone in 18%, anaemia with leucopenia in 11%, and anaemia with thrombocytopenia in 14%. The most common symptom cluster was pallor with fatigue, bleeding, weight loss, and jaundice, seen in 37% of patients. Pallor with fatigue and bleeding occurred in 19%, and pallor with fatigue, jaundice, and weight loss in 14%. Only 1% presented with fatigue and bleeding, and 2% with weight loss alone. The study did not report survival, treatment outcomes, or response to any specific therapy.
A single case report described a 57-year-old man who developed recurrent anaemia and lower-limb paraesthesia four years after small bowel resection. Peripheral blood film and bone marrow cytology showed megaloblastic changes. His anaemia and paraesthesia resolved with parenteral cyanocobalamin. No other patients were included, and no generalisable efficacy data were provided.
Across these studies, no randomised trial, no controlled comparison of treatments, and no long-term follow-up data exist. What is missing is a prospective trial that stratifies patients by underlying cause—nutritional deficiency, malabsorption, or post-surgical state—and measures haematological recovery, neurological outcomes, and relapse rates with standardised vitamin B12 and folate replacement. Funding for such a trial and a design that accounts for the heterogeneity of megaloblastic anaemia remain absent.
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 · 1980 · 56 citations
Congenital methylmalonic aciduria--homocystinuria with megaloblastic anemia: observations on response to hydroxocobalamin and on the effect of homocysteine and methionine on the deoxyuridine suppression test
AbstractAbstract Congenital methylmalonic aciduria-homocystinuria, a disorder with an incompletely defined cobalamin abnormality, has not been accompanied by megaloblastosis in most of the initially described cases. This has raised questions about the exact role of cobalamin in relation to megaloblastic anemia. Therefore, we present our observations on a patient with this syndrome whose manifestations conformed to current concepts of cobalamin function and on her response to hydroxocobalamin. In addition to the megaloblastic anemia (and other hematologic abnormalities) she developed severe neurologic impairment shortly after birth. Hydroxocobalamin injections diminished her methylmalonic acid and homocys-tine excretion and corrected her megaloblastosis, though mild neutrophil nuclear hypersegmentation has persisted even after a year. Clinical status also improved, particularly physical growth and neurologic development. However, she remains neurologically impaired following an unexplained clinical catastrophe. We documented her megaloblastic process by demonstrating abnormal deoxyuridine suppression test results in her initial bone marrow aspirate and normal results after treatment. Furthermore, the abnormal deoxyuridine suppression was corrected in vitro by folic acid, but not by methyltetrahydrofolic acid, cyanocobalamin, adenosylcobalamin, methylcobalamin, or methionine. Homocysteine slightly worsened the deoxyuridine suppression pattern. Subsequent studies showed that, in contrast to previous reports, homocysteine had no beneficial effect in other normoblastic or megaloblastic marrows either. The findings in our patient are compatible with the “methyltetrahydrofolate trap” hypothesis. Why this syndrome presents such a wide spectrum of manifestations is not entirely clear, nor is it clear why it should differ so from other disorders of cobalamin. These aspects are reviewed. Severity of the defect, differences in tissue susceptibility, or possibly even other factors such as ability to retain cobalamin intracellularly may influence the manifestations of this metabolic disorder.
Medical Journal of Dr D Y Patil University · 2016 · 11 citations · open access
Megaloblastic anemia - A clinical spectrum and a hematological profile: The day-to-day public health problem
Abstract<b>Aims and Objectives:</b> To know the various parameters and diagnostic approach of megaloblastic anemia. To know the age incidence and sex ratio. <b>Materials and Methods:</b> A hospital-based retrospective and prospective study was done for a period of 1-year. Totally, 21 cases were analyzed, and we correlated signs, symptoms, and hematological investigations. <b>Results:</b> Totally, 21 children with megaloblastic anemia in the above said period were studied. The patients age group was ranged from 2 months to 15 years. Megaloblastic anemia was observed in all the cases. <b>Conclusion:</b> Megaloblastic anemia is one of the common causes of undiagnosed anemia, and the treatment is simple and easily affordable. If left untreated, it can lead to morbidity both because of anemia and attendant neurological involvement.
The Professional Medical Journal · 2023 · 1 citations · open access
Study of clinico-pathological profile in patients with megaloblastic anemia.
AbstractObjective: To determine the clinico-pathological profile in patients with megaloblastic anemia. Study Design: Cross Sectional study. Setting: Department of Pathology, Sheikh Zayed Medical College/Hospital, Rahim Yar Khan. Study Period: June 2022 to July 2023. Material & Methods: The study subjects were 100 patients suffering from megaloblastic anemia. Using convenient sampling technique, blood samples were analyzed for complete blood count (CBC) and peripheral blood morphology followed by serum B12, serum and red cell folate levels and the bone marrow examination. SPSS version 25 was utilized to analyze the data. The results of variables including age, gender, clinical features and hematological profile were analyzed. Results: Out of 100 diagnosed patients of megaloblastic anemia, 51 (51%) were males, whereas 49 (49%) were females. Anemia alone was present in 18(18%), 11(11%) had anemia along with leucopenia, 14 (14%) faced anemia with thrombocytopenia, 57(57%) had pancytopenia while 16(16%) were pallor. Moreover, 01(01%) of the studied patients presented with fatigue and bleeding while 02 (2%) got weight loss. 10(10%) had fatigue accompanying with Pallor. 19(19%) faced pallor associated with fatigue and bleeding. 14(14%) had pallor accompanying with fatigue, Jaundice and weight loss. 37(37%) had pallor along with fatigue, bleeding, weight loss and jaundice. Conclusion: Males were more frequently affected by megaloblastic anaemia than females. The frequent clinical features appreciated were fatigue, pallor, weight loss, bleeding, and jaundice. Common cytopenia was pancytopenia, followed by bicytopenia.
Nigerian Journal of Medicine · 2021 · 0 citations · open access
Megaloblastic Anemia Associated with Small Bowel Resection in an Adult Patient
AbstractMegaloblastic anemia is characterized by macro-ovalocytosis, cytopenias, and nucleocytoplasmic maturation asynchrony of marrow erythroblast. The development of megaloblastic anemia is usually insidious in onset, and symptoms are present only in severely anemic patients. We managed a 57-year-old male who presented at the Hematology clinic on account of recurrent anemia associated with paraesthesia involving the lower limbs, 4-years-post small bowel resection. Peripheral blood film and bone marrow cytology revealed megaloblastic changes. The anemia and paraesthesia resolved with parenteral cyanocobalamin.
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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