News

A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial…

Intensifying treatment with hydroxyurea in young children with sickle cell anemia is safe and improves several disease parameters in the long run, researchers demonstrated in a clinical trial (NCT00305175) comparing toddlers with older children. Based on the positive findings, researchers argue that the practice of giving small children higher…

The Bristol-Myers Squibb Foundation, Texas Children’s Cancer and Hematology Centers and Baylor College of Medicine plan to jointly create a treatment network for children with cancer and blood diseases in three sub-Saharan African countries. The $100 million initiative, Global HOPE (Hematology-Oncology Pediatric Excellence), aims to build the medical capacity needed to diagnose and properly…

Modus Therapeutics, a company developing treatments specifically for sickle cell disease, has successfully completed a financing campaign that raised a total of 32 million Swedish krona (about €3.4 million euros) to develop sevuparin. Sevuparin is a proprietary polysaccharide drug with the potential to restore blood flow and prevent…

Researchers in Alicante, Spain, established levels of normality for hemoglobin A in newborn screening by taking into account factors such as the babies being born prematurely and their mothers having immigrated from an area of the world where the prevalence of genetics mutations causing defects in the hemoglobin gene is high.

Using stem cells from a closely matched sibling increases the chance that a sickle-cell disease transplant will succeed, and the patient survive, a study reports. The study, “Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation,” was published in the journal Blood. It…

Hemoglobin A1c (HbA1c), a biomarker that measures blood sugar levels, may systematically underestimate past glycemia among African-American patients with sickle cell trait (SCT). These findings highlight the importance of further evaluating this biomarker, which may be leading to misdiagnoses and poor treatment among black patients. The study “Association of…

The National Institutes of Health (NIH) has awarded a $4.3 million research grant to Dr. Allison A. King of the Washington University School of Medicine in St. Louis to support her research on sickle cell disease, with particular focus on teens and adults. The grant — believed to be the…

Genetically engineered mini-pig models of sickle cell disease (SCD) that closely mimic the condition in people will be created as part of a contract with Exemplar Genetics. The models could help researchers find new treatments for the disease. The work will support the efforts of the National Institutes of…

The β-hemoglobin gene and protein in humans and mice are similar, which makes a laboratory mouse a very useful partner in studies of β-hemoglobin-related disorders, like sickle cell disease, and possible treatment targets. A review looked at the benefits and shortcomings of animal models of β-hemoglobin diseases, and suggested that the time had come to move “beyond” them.