Using a CRISPR-based screening tool, Children's Hospital of Philadelphia researchers identified the signaling protein HRI, or heme-regulated eIF2alpha kinase, as a regulator of fetal hemoglobin in red blood cells. Suppression of HRI in cultures of red blood cells resulted in increased production of fetal hemoglobin, according to the study in the journal Science. Higher levels of HbF have been associated with reduced rates of acute painful episodes and reduced severity of sickle cell disease.
The half-life of prophylactic factor VIIa variant marzeptacog alfa (activated) was extended by six hours when administered subcutaneously in a Phase II/III trial involving patients with hemophilia A or B with inhibitors. The findings were presented at a meeting of the International Society on Thrombosis and Haemostasis.
Akers Biosciences has signed Appleton Medical Services to market the PIFA Heparin/PF4 Rapid Assay, a heparin-induced thrombocytopenia test.
Researchers from the New York Stem Cell Foundation Research Institute have developed a bone engineering technique called SATE, or Segmental Additive Tissue Engineering. Details of the process, which involves bone sections engineered into a graft using stem cells, were published in the journal Scientific Reports.
Cellectar Biosciences announced that a midstage trial of the investigative drug CLR 131 found an overall response rate of 33% among patients with B-cell cancers and a disease control rate of 50%, with tumor reduction rates from 60% to more than 90%. A patient with Waldenstrom macroglobulinemia experienced a 94% tumor reduction after two doses.
A study found community-based malaria interventions in western Kenya, including free testing and discounts on artemisinin-based treatment, were associated with greater use of first-line antimalarial drugs by patients who needed them. The findings were reported in the journal PLOS Medicine.
An international group of researchers has created an early warning system intended to alert authorities in Barbados before potential dengue outbreaks based on meteorological data. Another system was previously developed for Ecuador, and the idea could be used more widely if tests show it works, said Rachel Lowe of the London School of Hygiene and Tropical Medicine.
House Ways and Means Committee Chairman Kevin Brady, R-Texas, said he is talking with HHS Secretary Alex Azar about how to resume Affordable Care Act risk adjustment payments after the administration suspended them earlier this month. House lawmakers intend to consider a number of health care bills next week, including legislation to expand health savings accounts and postpone or repeal certain Affordable Care Act taxes, but Brady said he's not sure if a risk adjustment fix will be among the measures.
Patients with IDH1-mutated relapsed or refractory acute myeloid leukemia were found to be responsive to Agios Pharmaceuticals' ivosidenib based on data published in the New England Journal of Medicine that showed the drug produced durable remissions among the 125 study participants. The cohort was administered 500 mg of ivosidenib daily with a minimum follow-up of six months.
ThermoGenesis, a subsidiary of Cesca Therapeutics, has launched its PXP System for the processing of autologous bone marrow cells at the point of care.
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