Fighting sickle cell disease using a type 2 diabetes medication
Dr. Vivien Sheehan, assistant professor of pediatrics at Baylor and Texas Childrens Cancer and Hematology Centers
Sickle cell disease and the blood disorder beta thalassemia affect more than 180,000 Americans and millions more worldwide. Both diseases can be made milder or even cured by increasing fetal hemoglobin (HbF) levels, but current treatment to ramp up HbF is limited in its effectiveness. Researchers at Baylor College of Medicine and Texas Childrens Cancer and Hematology Centers have discovered a gene, FOXO3, involved in controlling fetal hemoglobin production and were able to target the gene and turn on fetal hemoglobin levels in patient samples in the lab using the diabetes drug metformin. This offers promising new treatments the first new drug treatment for sickle cell disease in 30 years and the first ever for beta thalassemia.
It was a major breakthrough to show that a common drug already in use for type 2 diabetes could be a treatment for sickle cell disease by inducing fetal hemoglobin, a type of hemoglobin that doesnt become sickle shaped but is usually turned off in infancy, said Dr. Vivien Sheehan , assistant professor of pediatrics at Baylor and Texas Childrens Cancer and Hematology Centers and lead investigator of the research. This is an exciting example of collaborative, bench-to-bedside research that has now resulted in a clinical trial that is already enrolling patients.
Sheehan launched this research as a clinical fellow at Baylor College of Medicine in 2011 with the goal of identifying new drug targets to help sickle cell patients make more f Continue reading