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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At-home measurement of WBCs

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Red and white blood cells
A portable device could be used to monitor patients’ white blood cell (WBC) levels at home, without the need for blood samples, according to researchers. The team created a prototype that records video of blood cells flowing through capillaries just below the skin surface at the base of the... [Read Article]
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How RBCs maintain their shape

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From left to right:
Study authors Velia
Fowler, Alyson Smith,
and Roberta Nowak
Photo by Don Boomer
New research indicates that non-muscle myosin II-A (NMIIA) plays a key role in maintaining red blood cell (RBC) shape and deformability. Researchers found evidence to suggest that NMIIA forms filaments in RBCs, and specialized regions at both... [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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Drug nets orphan designation for SCD

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A sickled red blood cell
beside a normal one
Image by Betty Pace
The European Commission (EC) has granted orphan designation to Altemia (formerly SC411) for the treatment of pediatric patients with sickle cell disease (SCD). Altemia gelatin capsules are designed to replenish the lipids destroyed by sickle hemoglobin. Altemia is intended to be... [Read Article]
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Test for HIT receives CE mark

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Platelet-rich plasma
HIT Confirm, a test used to diagnose heparin-induced thrombocytopenia (HIT), has received the CE mark. This means the test meets regulatory requirements and health, safety, and environmental protection standards for products sold within the European Economic Area. HIT Confirm is a flow cytometry-based test developed by Emosis. The test can be... [Read Article]
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Team uses CRISPR to turn on fetal hemoglobin

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Merlin Crossley
Photo from UNSW
Researchers have used CRISPR-Cas9 gene editing to reproduce naturally occurring mutations that boost the production of fetal hemoglobin. The mutations are associated with hereditary persistence of fetal hemoglobin (HPFH), and the researchers believe that introducing these mutations into erythroid cells could be a safe way to treat β-hemoglobinopathies such as... [Read Article]
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