DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for acute myelomonocytic leukemia — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAcute myelomonocytic leukemia maps to a 1-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
approvedDaunorubicinApproved drugapprovedEtoposideDNA topoisomerase II alpha inhibitor
Structures already discussed alongside acute myelomonocytic leukemia in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
Human topoisomerase IIbeta — Etoposide has a real, experimentally solved structure in complex with this target (PDB 3QX3, 2.162 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 evpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3QX3 · 2.162 Å · ligand Etoposide (EVP). Experimental structure, not a prediction.
What the evidence adds up to
No abstract in this set reports a clinical trial or patient outcome for acute myelomonocytic leukaemia specifically. The 2013 consensus document on chronic myelomonocytic leukaemia (CMML) recommends that patients with myelodysplastic-type CMML and less than 10% bone marrow blasts receive supportive therapy to correct cytopenias; those with 10% or more blasts should add 5-azacytidine. For myeloproliferative-type CMML with low blasts, hydroxyurea is the drug of choice to control proliferation and reduce organomegaly; patients with high blasts should receive polychemotherapy. Allogeneic stem cell transplantation is recommended only within clinical trials for selected patients. No response rates or survival figures are given for any of these approaches.
The 2021 review of novel therapies in acute myeloid leukaemia (AML) notes that nine agents have been approved since 2017, but it does not name any drug that has been tested in acute myelomonocytic leukaemia or report any outcomes for that subtype. The 2020 book and the 2025 rational pharmacotherapy paper discuss AML and chronic leukaemias in general terms, listing agents such as anthracyclines, cytarabine, tyrosine kinase inhibitors, and BCL-2 inhibitors, but again provide no data specific to acute myelomonocytic leukaemia.
What is missing is any prospective trial or retrospective analysis that isolates acute myelomonocytic leukaemia as a distinct cohort. No abstract gives a sample size, response rate, or survival statistic for this disease. Without dedicated trials that stratify patients by the specific French-American-British or WHO subtype, the evidence base remains extrapolation from broader AML or CMML studies. Funding for a subtype-specific trial, a validated biomarker to select patients, and a consensus on whether acute myelomonocytic leukaemia should be treated as AML or as CMML are all 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 · 2002 · 48 citations · open access
Results of consecutive trials for children newly diagnosed with acute myeloid leukemia from the Australian and New Zealand Children's Cancer Study Group
AbstractDespite improvements in the treatment of acute myeloid leukemia (AML), approximately 50% of children die of the disease. Clinical trials in adult patients with AML indicate that idarubicin may have superior efficacy when compared to daunorubicin in the remission-induction phases of chemotherapy. We conducted consecutive clinical trials in children with newly diagnosed AML in which daunorubicin (group 1, n = 102) or idarubicin (group 2, n = 160) was used during the remission-induction (RI) and the early consolidation phases of chemotherapy. Idarubicin was given at a dose of either 10 mg/m(2) (group 2A, n = 106) or 12 mg/m(2) (group 2B, n = 53). A high rate of RI was achieved for all groups (95% group 1, 90% group 2A, 94% group 2B). There were no significant differences in 5-year event-free survival (EFS) or in overall survival (OS) when the 3 groups were compared (group 1: EFS 50%, OS 56%; group 2A: EFS 50%, OS 60%; group 2B: EFS 34%, OS 50%). RI deaths resulting from treatment toxicity were low-2% for group 1 and 5% for group 2. More gastrointestinal, pulmonary, and renal toxicity but fewer infections were observed in patients receiving idarubicin (P <.001, P =.04, P =.03, respectively). Following RI chemotherapy, all patients received 3 to 4 more courses of identical chemotherapy and then underwent either autologous (n = 156) or an allogeneic bone marrow transplantation (BMT) (n = 35). OS was higher in allogeneic BMT patients than in autologous BMT patients (79% vs 63%; P =.23). We conclude that daunorubicin is as effective as idarubicin for remission-induction therapy for childhood AML and has reduced toxicity.
Management recommendations for chronic myelomonocytic leukemia: consensus statements from the SIE, SIES, GITMO groups
AbstractWith the aim of reviewing critical concepts and producing recommendations for the management of chronic myelomonocytic leukemia, key questions were selected according to the criterion of clinical relevance. Recommendations were produced using a Delphi process and four consensus conferences involving a panel of experts appointed by the Italian Society of Hematology and affiliated societies. This report presents the final statements and recommendations, covering patient evaluation at diagnosis, diagnostic criteria, risk classification, first-line therapy, monitoring, second-line therapy and allogeneic stem cell transplantation. For the first-line therapy, the panel recommended that patients with myelodysplastic-type chronic myelomonocytic leukemia and less than 10% blasts in bone marrow should be managed with supportive therapy aimed at correcting cytopenias. In patients with myelodysplastic-type chronic myelomonocytic leukemia with a high number of blasts in bone marrow (≥ 10%), supportive therapy should be integrated with the use of 5-azacytidine. Patients with myeloproliferative-type chronic myelomonocytic leukemia with a low number of blasts (<10%) should be treated with cytoreductive therapy. Hydroxyurea is the drug of choice to control cell proliferation and to reduce organomegaly. Patients with myeloproliferative-type chronic myelomonocytic leukemia, and a high number of blasts should receive polychemotherapy. Both in myelodysplastic-type and myeloproliferative-type chronic myelomonocytic leukemia, allogeneic stem cell transplantation should be offered within clinical trials in selected patients.
Leukemia & lymphoma/Leukemia and lymphoma · 1993 · 4 citations
Application of Low-Dose Etoposide Therapy for Myelodysplasia Syndromes
AbstractThe therapy for myelodysplastic syndromes (MDS) and acute leukemia (AL) transformed from MDS is not well established. Etoposide (VP 16-213) at low concentrations shows differentiation-inducing activity against leukemic cells in vitro. A prior study showed that oral low-dose etoposide therapy was effective in patients with chronic myelomonocytic leukemia. We used low-dose etoposide to treat six patients with refractory anemia with excess blasts in transformation (RAEB-t) and seven patients with AL transformed from MDS. The etoposide (50 mg, 2-7 times/week) was usually administered intravenously to ensure reliable bioavailability. Of 12 assessable patients, four RAEB-t patients achieved a partial response and one AL patient achieved complete remission. The responders became transfusion-independent, and this continued for 2-9 months while etoposide therapy was continued. Three of five responders had been resistant to prior repeated low-dose cytarabine therapy. The side effects were mild and well tolerated. Heterogeneous mechanisms were surmised to explain the clinical effects of low-dose etoposide. Several aspects, including the optimal schedule of low-dose etoposide therapy, the effect of this therapy on the patients' survival, the usefulness of combination therapy with other chemotherapeutic drug(s) and/or cytokine(s), should be investigated in the future.
Leukemia & lymphoma/Leukemia and lymphoma · 2021 · 3 citations · open access
Incorporation of Novel therapies for the treatment of acute myeloid leukemia: a perspective
AbstractAcute myeloid leukemia (AML) is a heterogeneous group of diseases that poses an array of therapeutic challenges. For decades two chemotherapeutic agents, cytarabine and daunorubicin, remained the backbone of AML therapy protocols. However, since 2017 nine novel therapies have been approved for the management of AML. With the rapid expansion of therapeutic options, hematologists must adapt their practice to optimize the benefits of these novel therapy options and minimize treatment toxicity. Here, we discuss the novel therapies that have changed the standard of care in management of patients with AML. We summarize the pivotal clinical trials that lead to the approval of these agents, and ongoing trials evaluating additional potential indications. We discuss several promising therapy candidates and their corresponding clinical trials. We discuss therapeutic strategies to incorporate these therapies into practice and pose unanswered questions that have arisen along with the expansion of treatment options.
IntechOpen eBooks · 2020 · 0 citations · open access
Acute Leukemias
AbstractWritten by expert research teams, this book describes different aspects of both acute myeloid and acute lymphocytic leukemia, specifically their pathobiology, classification/diagnosis, and treatment. Chapters highlight current research as well as the gold standards for diagnosis and treatment of these diseases, examining recent advances in personalized approaches to acute leukemia.
Zenodo (CERN European Organization for Nuclear Research) · 2025 · 0 citations · open access
Rational pharmacotheray in acute and chronic leukemia
AbstractLeukemia, encompassing acute and chronic forms, is a heterogeneous group of hematological malignancies characterized by uncontrolled proliferation of immature or mature leukocytes. Rational pharmacotherapy aims to optimize treatment outcomes through evidence-based drug selection, tailored regimens, and minimization of toxicity. In acute leukemias (acute lymphoblastic leukemia [ALL] and acute myeloid leukemia [AML]), pharmacotherapy focuses on intensive multi-agent chemotherapy for induction of remission, followed by consolidation and maintenance phases. Agents such as anthracyclines, cytarabine, methotrexate, and vincristine are widely employed, with newer targeted therapies like tyrosine kinase inhibitors (TKIs) and monoclonal antibodies enhancing remission rates, particularly in Philadelphia chromosome-positive ALL and FLT3-mutated AML. In chronic leukemias (chronic myeloid leukemia [CML] and chronic lymphocytic leukemia [CLL]), long-term disease control rather than immediate cure is the primary goal. TKIs (imatinib, dasatinib, nilotinib) revolutionized CML management, transforming it into a chronic controllable disease. In CLL, agents such as Bruton’s tyrosine kinase inhibitors (ibrutinib), BCL-2 inhibitors (venetoclax), and anti-CD20 monoclonal antibodies (rituximab, obinutuzumab) provide superior efficacy compared with traditional chemotherapy. Supportive pharmacotherapy—including antimicrobials, hematopoietic growth factors, and transfusion support—plays a critical role in reducing complications and improving quality of life. Emerging precision medicine approaches, such as CAR-T cell therapy and molecularly guided regimens, further expand therapeutic possibilities. Rational pharmacotherapy in leukemia thus integrates conventional cytotoxic agents, targeted therapies, and supportive care, with individualized treatment plans based on leukemia subtype, genetic profile, disease stage, and patient-specific factors, to maximize survival and minimize adverse outcomes.
Zenodo (CERN European Organization for Nuclear Research) · 2025 · 0 citations · open access
Rational pharmacotheray in acute and chronic leukemia
AbstractLeukemia, encompassing acute and chronic forms, is a heterogeneous group of hematological malignancies characterized by uncontrolled proliferation of immature or mature leukocytes. Rational pharmacotherapy aims to optimize treatment outcomes through evidence-based drug selection, tailored regimens, and minimization of toxicity. In acute leukemias (acute lymphoblastic leukemia [ALL] and acute myeloid leukemia [AML]), pharmacotherapy focuses on intensive multi-agent chemotherapy for induction of remission, followed by consolidation and maintenance phases. Agents such as anthracyclines, cytarabine, methotrexate, and vincristine are widely employed, with newer targeted therapies like tyrosine kinase inhibitors (TKIs) and monoclonal antibodies enhancing remission rates, particularly in Philadelphia chromosome-positive ALL and FLT3-mutated AML. In chronic leukemias (chronic myeloid leukemia [CML] and chronic lymphocytic leukemia [CLL]), long-term disease control rather than immediate cure is the primary goal. TKIs (imatinib, dasatinib, nilotinib) revolutionized CML management, transforming it into a chronic controllable disease. In CLL, agents such as Bruton’s tyrosine kinase inhibitors (ibrutinib), BCL-2 inhibitors (venetoclax), and anti-CD20 monoclonal antibodies (rituximab, obinutuzumab) provide superior efficacy compared with traditional chemotherapy. Supportive pharmacotherapy—including antimicrobials, hematopoietic growth factors, and transfusion support—plays a critical role in reducing complications and improving quality of life. Emerging precision medicine approaches, such as CAR-T cell therapy and molecularly guided regimens, further expand therapeutic possibilities. Rational pharmacotherapy in leukemia thus integrates conventional cytotoxic agents, targeted therapies, and supportive care, with individualized treatment plans based on leukemia subtype, genetic profile, disease stage, and patient-specific factors, to maximize survival and minimize adverse outcomes.
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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