 | | Vol. 12.25 – 29 June, 2021 |
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| Scientists assessed the safety and clinical activity of ciltacabtagene autoleucel, a chimeric antigen receptor T-cell therapy with two B-cell maturation antigen-targeting single-domain antibodies, in patients with relapsed or refractory multiple myeloma with poor prognosis.
[Lancet] |
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 | | PUBLICATIONSRanked by the impact factor of the journal |
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| Researchers described the stepwise evolution of gilteritinib resistance in FLT3-mutated acute myeloid leukemia (AML). Early resistant cells underwent metabolic reprogramming, grew more slowly, and were dependent upon Aurora kinase B.
[Cancer Cell] |
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| Investigators identified and validated the presence of hematopoietic stem and progenitor cells (HSPCs) within glioblastoma tissues, and demonstrated a positive link of tumor-associated HSPCs with malignant and immunosuppressive phenotypes.
[Nature Communications] |
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| Using single-cell RNA sequencing, the authors delineated the transcriptional heterogeneity of human bone marrow endothelium, showing increased expression of inflammatory genes, including IL1B and MYC, in ischemic heart failure.
[Nature Communications] |
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| Researchers demonstrated that genotoxic vectors induced a unique gene expression signature linked to stemness and oncogenesis in transduced murine hematopoietic stem- and progenitor cells, and developed the Surrogate Assay for Genotoxicity Assessment.
[Molecular Therapy] |
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| Investigators demonstrated that their robust, patient-derived xenograft (PDX) model for myelodysplastic syndrome was applicable as a preclinical platform to address pending clinical questions by interrogating the efficacy and safety of the thrombopoietin receptor agonist eltrombopag.
[Leukemia] |
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| Using diffuse large B cell lymphoma cell line models, the authors demonstrated the tumor suppressor activity of Germinal Center Proliferative Adapter RNA (GECPAR), which was mediated via its transcriptional regulation of proliferation and differentiation genes, particularly MYC and the Wnt pathways.
[Haematologica] |
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| By implementing pharmacogenomics and a cell-based drug screen, scientists found that THZ531 led to inhibition of oncogenic transcriptional programs, especially the DNA damage response pathway, MYC target genes and the mTOR-4EBP1-MCL-1 axis, contributing to dramatic lymphoma suppression in vitro.
[Haematologica] |
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| Investigators conducted a pilot clinical trial of a humanized CD19 CAR T-cell product in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia or B-lymphoblastic lymphoma, treated in two cohorts: with or without prior CAR exposure.
[Journal of Clinical Oncology] |
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| 120 patients with de novo acute myeloid leukemia received either romyelocel-L infusion on day nine with granulocyte colony-stimulating factor (G-CSF) starting daily on day fourteen (treatment group) or G-CSF daily alone on day fourteen (control) until absolute neutrophil count recovery to 500/µL.
[Journal of Clinical Oncology] |
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| Researchers conducted a Phase I clinical trial of H3B-8800, an oral small molecule that bound Splicing Factor 3B1 (SF3B1), in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, or acute myeloid leukemia.
[Leukemia] |
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| Scientists enrolled 38 patients with hematologic malignancies scheduled to undergo MMUD-HCT onto one of two conditioning strata: myeloablative using fludarabine and fractionated total body irradiation or reduced-intensity with fludarabine/melphalan.
[Blood Advances] |
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| Zanubrutinib, a highly selective Bruton tyrosine kinase inhibitor, was evaluated in a Phase I/II study in patients with various B-cell malignancies. Zanubrutinib was well tolerated and demonstrated activity in patients with relapsed/refractory mantle cell lymphoma.
[Blood Advances] |
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| The authors review the molecular effects of splicing factor mutations on splicing, mechanisms by which these mutations drive clonal transformation of hematopoietic cells, and the development of new therapeutics targeting these genetic subsets of hematopoietic malignancies.
[Blood] |
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| Scientists review the role of the cohesin complex in healthy and malignant hematopoiesis and discuss clinical implications of cohesin mutations in myeloid malignancies and opportunities for therapeutic targeting.
[Blood] |
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| Investigators discuss recent therapeutic advances and innovative treatment strategies in chronic myelomonocytic leukemia (CMML), including global and molecularly targeted approaches, and discuss what may help to make progress in the design of rationally derived and disease-modifying therapies for CMML.
[Leukemia] |
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| Imara, Inc. announced the grant recipients of its second annual Real Impact community support initiative. This program, which includes grant funding to support nonprofit, community-based organizations (CBOs) serving patients and families impacted by sickle cell disease and beta-thalassemia, awarded 30 grants to CBOs in 16 states totaling $150,000.
[Imara, Inc.] |
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| HUYABIO International™ announced the regulatory approval for HBI-8000 monotherapy of relapsed or refractory adult T-cell leukemia/lymphoma (ATL) by the Japanese Pharmaceuticals and Medical Devices Agency. The drug was approved based on data from a Phase IIb study that involved 23 patients with aggressive ATL in Japan.
[HUYABIO International™] |
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| BioCardia®, Inc. announced that the independent Data Safety Monitoring Board has completed its prespecified data review for the Phase III pivotal CardiAMP™ Cell Therapy Heart Failure Trial. CardiAMP cell therapy uses a patient’s autologous bone marrow cells delivered to the heart in a minimally-invasive procedure.
[BioCardia®, Inc.] |
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| I-Mab Biopharma Co., Ltd. announced that the Center for Drug Evaluation of the China National Medical Products Administration (NMPA) has approved the Investigational New Drug application to initiate a phase Ib trial of felzartamab, a CD38 antibody, in patients with systemic lupus erythematosus.
[I-Mab Biopharma Co., Ltd.] |
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| | Heinrich Heine University Düsseldorf – Düsseldorf, Germany |
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| | St. Jude Children’s Research Hospital – Memphis, Tennessee, United States |
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| | Cyprus Cancer Research Institute – Nicosia, Cyprus |
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| | Institute of Oncology Research – Bellinzona, Switzerland |
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| | University of Wisconsin Madison – Madison, Wisconsin, United States |
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