DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for acute basophilic leukemia — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAcute basophilic leukemia maps to a 5-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
approvedPonatinibApproved drug
Structures already discussed alongside acute basophilic leukemia in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
AP24534, a Pan-BCR-ABL Inhibitor for Chronic Myeloid Leukemia, Potently Inhibits the T315I Mutant and Overcomes Mutation-Based Resistance — Ponatinib has a real, experimentally solved structure in complex with this target (PDB 3IK3, 1.9 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 0lidrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3IK3 · 1.9 Å · ligand Ponatinib (0LI). Experimental structure, not a prediction.
What the evidence adds up to
Acute basophilic leukemia is extremely rare, and the published literature contains no controlled trials or systematic treatment data for this specific diagnosis. A 1989 case report describes a 20-month-old child with acute nonlymphocytic leukemia showing basophilic differentiation and the cytogenetic abnormality t(9,11)(p22,q23). Immature blasts responded well to induction therapy with etoposide, but leukemic cells that were more differentiated toward basophils were refractory to that drug. Complete remission was eventually achieved with a conventional multidrug regimen, though the report gives no further detail on which drugs were used or the duration of remission.
A 2021 case of primary chronic basophilic leukemia in a 93-year-old woman presented with fatigue, leukocytosis, 43% basophils in peripheral blood, and 56% basophils in bone marrow. Next-generation sequencing revealed mutations in IDH2, DNMT3A, and BCOR, the first time NGS had been performed in primary chronic basophilic leukemia. The patient was palliated with hydroxyurea to lower white blood cell counts. This initially had a good effect, but she died two months after diagnosis. No other treatment was reported.
A 1959 review notes that basophils fall proportionately with the decline of leukocyte counts during Myleran, P32, and x-ray therapy of chronic myelogenous leukemia and myeloid metaplasia, but this observation concerns chronic myelogenous leukemia, not acute basophilic leukemia. The same review states that patients with acute leukemia almost always have relative and absolute decreases of circulating basophils, which suggests that acute basophilic leukemia was not a recognised or studied entity at that time.
What is still missing: any prospective trial or systematic case series for acute basophilic leukemia; no standard induction or salvage regimen has been established; the rarity of the disease means no dedicated funding or multi-centre collaboration exists; patient stratification by cytogenetic or molecular subtype has not been attempted; and the role of targeted agents against IDH2, DNMT3A, or BCOR mutations has not been tested in this disease.
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 · 1959 · 20 citations · open access
The Basophilic Granulocyte
AbstractAbstract 1. The history, morphology and technics of counting of basophils have been briefly reviewed. 2. A marked relative and absolute increase of basophils was always found in chronic myelogenous leukemia. Moderate relative and absolute increases of basophils usually occurred in myeloid metaplasias (including polycythemia vera with leukocytosis). In some cases of iron-deficiency, hemolytic and toxic anemias of long standing there may be moderate increases of blood basophils. 3. Marked relative and absolute decreases of basophils occurred in almost all cases of neutrophilic leukocytosis or leukemoid reaction, associated with infection, neoplasia, tissue necrosis or acute anemia. Patients with chronic lymphatic leukemia, monocytic leukemia or acute leukemia almost always have relative and absolute decreases of circulating basophils. 4. In our experience there is no evidence that basophils possess any peculiar radioresistant qualities. In general, the basophils fall proportionately with the decline of leukocyte counts during Myleran, P32 and x-ray therapy of chronic myelogenous leukemia and myeloid metaplasia. The suppressing action of these therapeutic agents seems to be on the proliferating blast cell. 5. The function of the basophils is not known, but it has been postulated that they might act as "heparinocytes," inhibiting clotting and stasis of blood and lymph in areas of inflammation. 6. There is suggestive evidence that the basophilopenia in cases of infection, neoplasia, tissue necrosis and acute anemia is analogous to the eosinophilopenia of the "stress reaction" mediated via the adrenal glands; however, it must be admitted that this has not been unequivocally established, and other mechanisms may play a role in controlling the levels of circulating basophils.
Case Reports in Oncology · 2021 · 8 citations · open access
Monitoring Ponatinib in a Child with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
AbstractPonatinib is a third-generation tyrosine kinase inhibitor (TKI) reported to show a higher efficacy for adult Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL) than other TKIs. However, few studies describe ponatinib for pediatric Ph+ALL; therefore, the efficacy, safety, and optimal dosage have not been determined. Here, we report a 3-year-old girl with Ph+ALL treated by a ponatinib-containing regimen with therapeutic drug monitoring in the plasma and cerebrospinal fluid (CSF). In our case, a ponatinib-containing regimen was able to keep minimal residual disease negative, and the pharmacokinetics (PKs) of plasma ponatinib resembled that previously reported in adults. Penetration to the CSF was extremely limited. Thus, ponatinib was feasible and effective for a child with Ph+ALL, although the plasma concentration of ponatinib varied significantly throughout the treatment. The appropriate dosage should be confirmed in a prospective trial, including a detailed PK study.
A monoclonal antibody reacting with human basophils
AbstractBsp-1 is an IgM murine monoclonal antibody raised against the human erythroblastic leukemia cell line (HEL) that reacts with basophils but not neutrophils or eosinophils. Western blotting techniques showed that Bsp-1 reacts with a 45-kilodalton surface antigen on HEL cells. The distribution of Bsp-1 antigen on leukemic cells is confined to a basophilic leukemia cell line, KU812, chronic myeloid leukemia with basophilia, and some cases of acute undifferentiated leukemia. Bsp-1 might therefore be a useful reagent for the study of basophil function and differentiation.
Hematology/Oncology and Stem Cell Therapy · 2015 · 3 citations · open access
Basophilia and megakaryoblastic differentiation in a case of acute myeloid leukemia
AbstractBasophilia is commonly associated with chronic myelogenous leukemia, notably in the accelerated phase or during blast crisis. It is also associated with other myeloproliferative neoplasms. However, its association with acute leukemia is very rare and is described in association with acute basophilic leukemia and few acute myeloid leukemias (AMLs) with recurrent genetic abnormalities such as t(6;9)(p23;q34). Herein, we describe the morphological features and discuss the differential diagnosis of a case of AML with the blasts showing previously unreported unusual combination of megakaryoblastic and basophilic differentiation along with peripheral blood and bone marrow basophilia.
American Journal of Hematology · 1989 · 2 citations
Acute nonlymphocytic leukemia with basophilic differentiation and t(9,11)(p22,q23) in a child
AbstractA 20-month-old child was treated for acute nonlymphocytic leukemia (ANLL) with basophilic differentiation. His leukemic cells also had the cytogenetic abnormality of t(9,11)(p22,q23). Although immature blasts responded well to induction therapy with etoposide, the leukemic cells that were more differentiated toward basophils were quite refractory to the drug. However, complete remission was finally achieved with a conventional multidrug regimen.
Annals of Hematology & Oncology · 2021 · 0 citations · open access
Basophils Unchained: A Rare Form of Leukemia
AbstractPrimary Chronic Basophilic Leukemia (CBL) is an extremely rare disease and thus knowledge of this disease remains limited. There are no universal diagnostic criteria nor has the underlying pathogenetic mechanism been elucidated. In this peculiar case, we present a 93-year-old woman with an acute symptomatology of fatigue compounded by a manifest leukocytosis with 43% basophils in peripheral blood. The bone marrow aspirate also showed basophilia, 56% specifically. Interestingly, Next Generation Sequencing (NGS) on the bone marrow sample revealed three possible driver mutations, namely in the IDH2, DNMT3A, and BCOR gene. To date, NGS has never been performed on samples of patients with primary CBL. Our findings could be of great value in the search for the pathogenetic mechanism of this rare disease and to help find a successful treatment. Our patient was palliated with hydroxyurea to lower the white blood cells. Initially, this had a good effect, but unfortunately, she died two months after the diagnosis.
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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