Familial risk of myeloid malignancies

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Three generations of
women in a family
A large study has revealed “the strongest evidence yet” supporting genetic susceptibility to myeloid malignancies, according to a researcher. The study showed that first-degree relatives of patients with myeloid malignancies had double the risk of developing a myeloid malignancy themselves, when compared to the general population. The researchers... [Read Article]
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FA pathway key to DNA repair after CRISPR cutting

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DNA repair
Image by Tom Ellenberger
New research suggests the Fanconi anemia (FA) pathway plays a key role in repairing double-strand breaks (DSBs) created by CRISPR-Cas9 genome editing. Researchers said they found that Cas9-induced single-strand template repair requires the FA pathway, and the protein FANCD2 localizes to Cas9-induced DSBs. The team said this research provides... [Read Article]
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CHMP recommends generic deferiprone

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Micrograph showing thalassemia
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for Deferiprone Lipomed to treat iron overload in patients with thalassemia major. Deferiprone Lipomed is a generic version of the iron chelating agent Ferriprox, which has been authorized in the European Union since August 1999. According to... [Read Article]
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CHMP backs 2 biosimilar pegfilgrastim products

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Neulasta Onpro Kit
Photo from Amgen
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for 2 pegfilgrastim biosimilar candidates—Udenyca and Pelgraz. Both products have been deemed highly similar to the reference product, Neulasta, a growth-colony-stimulating factor intended to reduce the duration of neutropenia and the incidence of febrile... [Read Article]
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Study could change treatment of MLSM7

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Study authors Tamara
Lamprecht, Jason Schwartz,
Jing Ma, and Jeffrey Klco
Photo from St. Jude
Children’s Research
Hospital/Justin Veneman
New findings could help improve treatment of an inherited bone marrow disorder known as myelodysplasia and leukemia syndrome with monosomy 7 (MLSM7), according to researchers. While studying families affected by MLSM7, researchers identified germline mutations... [Read Article]
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Gene therapy granted accelerated assessment

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DNA helix
Image by Spencer Phillips
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use has granted accelerated assessment for the upcoming marketing authorization application (MAA) for LentiGlobin™. LentiGlobin is a gene therapy intended for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype. bluebird bio intends... [Read Article]
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Treatments, disease affect spermatogonia in boys

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Male germinal epithelium
showing spermatogonia,
spermatocytes, spermatids,
and spermatozoa
Image from Dreamstime
Alkylating agents, hydroxyurea (HU), and certain non-malignant diseases can significantly deplete spermatogonial cell counts in young boys, according to research published in Human Reproduction. Boys who received alkylating agents to treat cancer had significantly lower spermatogonial cell counts than control subjects or boys... [Read Article]
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FDA approves biosimilar filgrastim

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Vials of drug
Photo by Bill Branson
The US Food and Drug Administration (FDA) has approved the leukocyte growth factor Nivestym™ (filgrastim-aafi), a biosimilar to Neupogen (filgrastim). Nivestym is approved to treat patients with nonmyeloid malignancies who are receiving myelosuppressive chemotherapy or undergoing bone marrow transplant, acute myeloid leukemia patients receiving induction or consolidation chemotherapy,... [Read Article]
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Kinase may be therapeutic target for hemoglobinopathies

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Gerd A. Blobel, MD, PhD
Photo from Children’s
Hospital of Philadelphia
A kinase called heme-regulated inhibitor (HRI) could be a therapeutic target for sickle cell disease (SCD) and some forms of β-thalassemia, according to researchers. The team found that reducing the activity of HRI (also known as EIF2AK1) can boost the production of fetal hemoglobin... [Read Article]
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L-glutamine reduces complications of SCD

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A sickled red blood cell
beside a normal one
Image by Betty Pace
Results of a phase 3 trial showed that L-glutamine can reduce complications of sickle cell disease (SCD). SCD patients who received pharmaceutical-grade L-glutamine (with or without hydroxyurea) had a reduction in sickle cell crises, hospitalizations, and acute chest syndrome (ACS) when compared... [Read Article]
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A new use for ibrutinib?

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Neutrophil engulfing bacteria
Image by Volker Brinkmann
Preclinical research suggests ibrutinib could treat G-CSFR-mutant myeloid disorders. “Mutations in G-CSFR have a harmful effect on the production of neutrophils and are reported in patients with several blood disorders, including severe congenital neutropenia, chronic neutrophilic leukemia, and acute myeloid leukemia,” said Ken Greis, PhD, of the University... [Read Article]
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FDA lifts hold on trial of MYC inhibitor

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Micrograph showing MDS
The US Food and Drug Administration (FDA) has lifted the clinical hold on a phase 1b trial of APTO-253. APTO-253 is a small molecule that inhibits expression of the c-Myc oncogene without causing general myelosuppression of the bone marrow, according to Aptose Biosciences Inc., the company developing the drug. Aptose was testing... [Read Article]
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Voxelotor benefits adolescents with SCD

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Attendees at the 23rd
Congress of the European
Hematology Association (EHA)
Photo from EHA
An ongoing phase 2 study suggests voxelotor (GBT440) can benefit adolescents with sickle cell disease (SCD). In the HOPE-KIDS 1 study, voxelotor produced sustained improvements in hemoglobin levels and a reduction in clinical measures of hemolysis in a cohort of adolescents... [Read Article]
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