Mon, 03/16/2015 – 3:58pm
Cynthia Fox, Science Editor
Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image (Credit: OpenStax College)A clinical trial of the most common sickle cell anemia drug, hydroxyurea, was halted a year early this winter because of “overwhelming evidence of benefit,” reported University of Nebraska Medical Center pediatric researcher Stephen Obaro, M.D., Ph.D., in The Lancet.Hydroxyurea is “the only readily available disease-modifying therapy for patients with sickle cell,” Principal Investigator Russell Ware, M.D., Ph.D., told Drug Discovery & Development, so this was good news to patients and clinicians. Ware is director of hematology at the Cincinnati Children’s Hospital.
“Recently published National Institutes of Health (NIH) evidence-based guidelines suggest it should be used more often,” Ware added.
But hydroxyurea does not solve all problems. It ultimately fails 50 percent of patients (if this number may drop with more widespread and knowledgeable use of the drug.) Average sickle cell patients only live into their 40s (if this number, too, could change with more widespread and knowledgeable use of the drug.) And while “ is widely available in the U.S. and Europe, it is not in low-resource countries,” Ware told Drug Discovery & Development. Given that two-thirds of the 305,800 newborns with sickle cell anemia live in Africa, this is one of many problems.
So other approaches are being investigated globally, including a pioneering genetically engineered stem cell clinical trial that recently enrolled its first patient.
Sickle cell anemia occurs as the result of an inherited mutation in the oxygen-carrying hemoglobin (Hbs) gene. This causes red blood cells to become sticky, and form a sickle shape, which impairs their ability to carry oxygen, and causes them to aggregate and form constant blockages in the blood stream. As red blood […]