Drug shows promise for treating AML, MDS

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Lab mouse
Preclinical results support clinical testing of an experimental agent in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), according to researchers. The agent, ALRN-6924, was shown to combat AML and MDS by restoring activity of the tumor-suppressing protein p53. ALRN-6924 exhibited antileukemic activity in AML cells and mouse models of the disease, as... [Read Article]
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Gene variants linked to survival after HSCT

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DNA helix
Image by Spencer Phillips
New research has revealed a link between rare gene variants and survival after hematopoietic stem cell transplant (HSCT). Researchers performed exome sequencing in nearly 2500 HSCT recipients and their matched, unrelated donors. The sequencing revealed several gene variants—in both donors and recipients—that were significantly associated with overall survival... [Read Article]
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Agent exhibits activity in leukemias, MDS

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AML cells
The experimental agent prexigebersen (formerly BP1001) was considered well-tolerated and demonstrated early evidence of activity against relapsed/refractory hematologic disorders in a phase 1/1b trial. The drug reduced blasts in the bone marrow and peripheral blood for patients with acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS). When given... [Read Article]
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Manufactured graft deemed safe in blood cancer patients

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Peripheral blood
stem cell graft
Phase 1 results suggest a programmed cellular therapy is safe for use in patients with hematologic malignancies. The therapy, ProTmune, is being developed as a next-generation allogeneic graft intended to reduce the incidence and severity of acute graft-versus-host disease (GVHD) after hematopoietic stem cell transplant (HSCT). Three of 7... [Read Article]
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Expanded UCB product can stand alone

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Mitchell Horwitz, MD
The expanded umbilical cord blood (UCB) product NiCord can be used as a stand-alone graft, according to research presented at the 2018 BMT Tandem Meetings. Researchers found that a single NiCord unit provided “robust” engraftment in a phase 1/2 study of patients with high-risk hematologic malignancies. NiCord recipients had quicker... [Read Article]
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NK-cell therapy in resistant MDS, AML

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Natural killer cell
destroying a cancer cell
Image by Joshua Stokes
Results of a phase 1/2 trial suggest treatment with haploidentical natural killer (NK) cells can be effective against relapsed/refractory myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). NK-cell therapy elicited responses in 6 of the 16 patients studied and provided a bridge... [Read Article]
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Azacitidine now available in China

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Vials of drug
Photo by Bill Branson
Azacitidine for injection (Vidaza®) is now available in China. The nucleoside metabolic inhibitor was approved in China to treat patients with intermediate-2/high-risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) with 20% to 30% bone marrow blasts, and chronic myelomonocytic leukemia (CMML). Azacitidine for injection is marketed in... [Read Article]
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Predicting response to AZA in MDS, CMML

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Micrograph showing MDS
Researchers have developed a technique that may help predict whether patients with myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) will respond to treatment with azacytidine (AZA). “The new method, called AZA-MS, utilizes a cutting-edge technique known as mass spectrometry to measure the different forms of AZA inside blood cells... [Read Article]
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