Patricia Inácio, PhD, science writer —

A non-invasive prenatal test to diagnose a risk of sickle cell disease in a baby is possible and may be available in clinics in a near future, if further testing confirms its efficacy, researchers in the U.K. report. Julia van Campen presented the first results of the non-invasive test in…

Treatment with hydroxyurea since early infancy, starting at the age of one, is effective and safe at preventing sickle cell disease (SCD) complications, a follow-up study has found. The study “Prospective longitudinal follow-up of children with sickle cell disease treated with hydroxyurea since infancy” was published in journal…

Imara’s investigational oral inhibitor IMR-687 is well-tolerated, and shows signs of potential for lowering blood biomarkers of sickle cell anemia (SCA), early data from a Phase 2 trial show. The results were presented at the 24th Congress of the European Hematology Association (EHA), held recently in…

The Phase 2 STRONG-SCD clinical trial evaluating olinciguat as an investigational therapy for sickle cell disease is seeking participants. Olinciguat, being developed by Cyclerion Therapeutics (a spin-off of Ironwood Pharmaceuticals), is an investigational oral therapy that stimulates an enzyme called soluble guanylate cyclase (sGC), known to play a key…

Adults with sickle cell disease score worse than their healthy, unaffected siblings on cognitive tests, despite treatment with hydroxyurea, a study reports. The study “Neurocognitive functioning in symptomatic adults with sickle cell disease: A description and comparison with unaffected siblings” was published in the journal Neuropsychological…

The success rate of bone marrow transplants for patients with sickle cell disease or beta thalassemia, from only “half-matched” donors, increased by doubling the radiation delivered to patients’ bodies before the transplants, a John Hopkins University study shows. The study, “Effect of increased dose of total body irradiation on…

A Phase 1/2 clinical trial evaluating investigational therapy CTX001 for severe sickle cell disease (SCD) has enrolled its first patient in the U.S., and treatment is expected to begin by mid-2019. The trial (NCT03745287), still recruiting participants in Nashville, Tennessee, is looking to enroll 45 adults, 18 to 35 years old,…

Sickle cell disease (SCD) patients were safely treated with a phosphodiesterase 9A (PDE9) inhibitor therapy called PF-04447943 with no serious side effects reported and possible evidence of fewer vaso-occlusion pain crises, a Phase 1b clinical trial reports. Safety and early signs of possible efficacy — including a drop in biomarkers linked…

The U.S. Food and Drug Administration (FDA) has awarded fast track status to investigational therapy CTX001 to treat sickle cell disease. A Phase 1/2 clinical trial (NCT03745287) is recruiting patients. CTX001 uses the CRISPR gene-editing technology to increase the production of fetal hemoglobin in patients’ red blood cells. Fetal hemoglobin is…

The amount of oxygen the blood loses when traveling through the brain could help identify children with sickle cell disease who are at risk of having a stroke, a study reports. Scientists call the measurement the oxygen extraction fraction because it refers to the percentage of oxygen that the brain…