2024
A Niche Driven Mechanism Determines Response to ATRA and a Mutation-Independent Therapeutic Approach for MDS and AML
Mosialou I, Ali A, Cuesta-Dominguez A, Labella R, Vgenopoulou V, Bisikirska B, Galan-Diez M, Wang A, Bewersdorf J, Liang C, Heiblig M, Jurcic J, Berman E, Rabadan R, Kornblau S, Garcia-Manero G, Raza A, Kousteni S. A Niche Driven Mechanism Determines Response to ATRA and a Mutation-Independent Therapeutic Approach for MDS and AML. Blood 2024, 144: 5788-5788. DOI: 10.1182/blood-2024-212307.Peer-Reviewed Original ResearchAcute myeloid leukemiaAcute myeloid leukemia patientsStandard of careComplete responseMyelodysplastic syndrome patientsMyelodysplastic syndromeOverall response rateResponse to ATRAAll-trans-retinoic acidB-cateninOsteoblast lineage cellsMyeloid malignanciesTreatment responseLineage cellsDuration of follow-upMonitoring of treatment responseResponse rateBone marrow biopsyAbsolute neutrophil countAdverse risk groupAcute myeloid leukemia cellsNotch signalingAssociated with complete inhibitionResponse to all-trans-retinoic acidResistance to standard
2023
E7820, an Anti-Cancer Sulfonamide, in Combination with Venetoclax in Patients with Splicing Factor Mutant Myeloid Malignancies: A Phase II Clinical Trial
Bewersdorf J, Chandhok N, Watts J, Derkach A, Abdel-Wahab O, Stein E, Taylor J. E7820, an Anti-Cancer Sulfonamide, in Combination with Venetoclax in Patients with Splicing Factor Mutant Myeloid Malignancies: A Phase II Clinical Trial. Blood 2023, 142: 1547. DOI: 10.1182/blood-2023-182369.Peer-Reviewed Original ResearchAcute myeloid leukemiaEastern Cooperative Oncology GroupPhase II clinical trialChronic myelomonocytic leukemiaMyelodysplastic syndromePatient-derived xenograftsII clinical trialsPreclinical dataSplicing factor genesMyeloid malignanciesClinical trialsAdequate end-organ functionContext of combination therapyDay 1Safety run-in phaseRefractory acute myeloid leukemiaSimon 2-stage designResponse rateDynamic BH3 profilingCycles of therapyNegative prognostic impactEvent-free survivalMyelodysplastic syndrome patientsHuman acute myeloid leukemiaPhase II trial