DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for sideroblastic anemia — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleSideroblastic anemia maps to a 3-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 sideroblastic 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
ATP binding cassette subfamily B member 7 (ABCB7) — ABCB7 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 atpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 23PI · 2.33 Å · ligand ADENOSINE-5'-TRIPHOSPHATE (ATP). Experimental structure, not a prediction.
What the evidence adds up to
Sideroblastic anemias are defined by ring sideroblasts — erythroid precursors with iron-loaded mitochondria that form a perinuclear ring. The condition can be hereditary, caused by mutations in genes such as ALAS2, ABCB7, or GRLX5, or acquired. Acquired forms include primary refractory anaemia with ring sideroblasts (RARS), a subtype of myelodysplastic syndrome, and secondary forms due to drugs, toxins, copper deficiency, or chronic neoplastic disease. The pathogenesis of mitochondrial iron loading varies: it can result from a defect in haem synthesis, in iron-sulfur cluster assembly, in release of iron-sulfur protein precursors from mitochondria, or in intracellular iron metabolism.
One 1995 letter reports a single 19-year-old man with sideroblastic anaemia who was treated with chloroquine. His haemoglobin was 5 g/dL at presentation, and his bone marrow contained 60 ringed sideroblasts per 100 erythroblasts. The letter states he was “successfully treated” but provides no numerical outcome data, no duration of response, and no control comparison. A 2023 case report describes a 20-year-old woman who developed isoniazid-induced sideroblastic anaemia while on antitubercular therapy without pyridoxine supplementation. Bone marrow studies confirmed the diagnosis. The report emphasises that this is a preventable cause of anaemia but gives no treatment outcome beyond noting the association.
In three patients with idiopathic sideroblastic anaemia who later developed acute leukaemia (two myeloblastic, one lymphoblastic), combination chemotherapy induced remission in all three. Remission durations were brief: 3, 2, and 3 months. Survival after leukaemia diagnosis was 13, 10, and 9 months. Remission inductions were complicated by prolonged bone marrow suppression. The ring sideroblast abnormality persisted during both leukaemic and remission phases, and transfusion requirements remained unchanged in the two patients who were transfusion-dependent throughout their course.
What is still missing are prospective trials with adequate sample sizes, standardised outcome measures, and stratification by genetic subtype. No therapy has been shown to durably correct the underlying mitochondrial iron loading or to reduce transfusion dependence in a controlled setting. The heterogeneity of the disorder — hereditary versus acquired, primary versus secondary — means that single-case reports and small series cannot support generalisable treatment recommendations.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Acta Haematologica · 2009 · 41 citations
Mitochondrial Iron Metabolism and Sideroblastic Anemia
AbstractSideroblastic anemias are a heterogeneous group of disorders, characterized by mitochondrial iron overload in developing red blood cells. The unifying characteristic of all sideroblastic anemias is the ring sideroblast, which is a pathological erythroid precursor containing excessive deposits of non-heme iron in mitochondria with perinuclear distribution creating a ring appearance. Sideroblastic anemias may be hereditary or acquired. Hereditary sideroblastic anemias are caused by defects in genes present on the X chromosome (mutations in the ALAS2, ABCB7, or GRLX5 gene), genes on autosomal chromosomes, or mitochondrial genes. Acquired sideroblastic anemias are either primary (refractory anemia with ring sideroblasts, RARS, representing one subtype of the myelodysplastic syndrome) or secondary due to some drugs, toxins, copper deficiency, or chronic neoplastic disease. The pathogenesis of mitochondrial iron loading in developing erythroblasts is diverse. Ring sideroblasts can develop as a result of a heme synthesis defect in erythroblasts (ALAS2 mutations), a defect in iron-sulfur cluster assembly, iron-sulfur protein precursor release from mitochondria (ABCB7 mutations), or by a defect in intracellular iron metabolism in erythroid cells (e.g. RARS).
Acute leukemia in idiopathic sideroblastic anemia: response to combination chemotherapy
AbstractAbstract Three patients with idiopathic sideroblastic anemia of variable duration developed acute leukemia. In two the leukemia was morphologically and histochemically myeloblastic, in one lymphoblastic. With combination chemotherapy remission was achieved in all three. The remission inductions were complicated by long periods of bone marrow suppression and the duration of remissions was brief (3, 2 and 3 months). Survival after diagnosis was 13, 10 and 9 mo, respectively. The ring sideroblast abnormality persisted during the leukemic and remission phases and transfusion requirements remained unaltered in the two patients with transfusion dependent anemia throughout their courses.
Acute leukemia in idiopathic sideroblastic anemia: response to combination chemotherapy
AbstractThree patients with idiopathic sideroblastic anemia of variable duration developed acute leukemia. In two the leukemia was morphologically and histochemically myeloblastic, in one lymphoblastic. With combination chemotherapy remission was achieved in all three. The remission inductions were complicated by long periods of bone marrow suppression and the duration of remissions was brief (3, 2 and 3 months). Survival after diagnosis was 13, 10 and 9 mo, respectively. The ring sideroblast abnormality persisted during the leukemic and remission phases and transfusion requirements remained unaltered in the two patients with transfusion dependent anemia throughout their courses.
Congenital sideroblastic anemia associated with germline polymorphisms reducing expression of FECH
AbstractThe sideroblastic anemias (SAs) are disorders of ineffective erythropoiesis, collectively characterized by abnormal Prussian blue-positive granules (i.e., iron-stuffed mitochondria) that encircle marrow erythroblast nuclei to form ringed sideroblast cells.[1][1] SAs are usually acquired, but
New England Journal of Medicine · 1995 · 8 citations
Treatment of Sideroblastic Anemia with Chloroquine
AbstractTo the Editor: The mainstay of treatment for sideroblastic anemia, a group of heterogeneous disorders1,2 involving a defect in heme synthesis,3 is transfusion. We describe a 19-year-old man with sideroblastic anemia who was successfully treated with chloroquine. The patient was referred to our institution in 1984 because of anemia. The white-cell count was 8.6×109 per liter, the platelet count 350×109 per liter, the hemoglobin level 5 g per deciliter, the mean cell volume 86 μm3, and the reticulocyte count 11×103 per liter. In the bone marrow, erythroid cellularity was normal, but there were 60 ringed sideroblasts per 100 . . .
International Journal of Research in Medical Sciences · 2023 · 0 citations · open access
A rare case of isoniazid induced sideroblastic anemia
AbstractSideroblastic anemia is a rare cause of anemia. Most of it accounts for the genetic cause, while drug induced is still uncommon. Our patient, a 20 year old female, is a known case of right frontal tuberculoma on ATT presented with complaints of generalized weakness and loss of appetite. On evaluation, she was found to have severe anemia and bone marrow studies confirmed it to be sideroblastic anemia. On revisiting the history, it was noted that she was not taking pyridoxine supplements as advised along with antitubercular drugs. Our patient is one among the few documented cases of Isoniazid induced sideroblastic anemia.This case needs attention because it is a preventable cause of anemia and the clinicians need to be aware about the compliance of the patient with the supplementary drugs.
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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