Scientists re-activate defective gene linked to sickle cell
Researchers from Baylor College of Medicine and the Texas Children’s Cancer and Hematology Centers recently discovered a new application for type 2 diabetes medication in the treatment of sickle cell disease.

The collaborative team identified the gene responsible for fetal hemoglobin production, and demonstrated a means by which the gene can be “turned on” using the diabetes drug metformin, thereby improving or sometimes curing conditions such as sickle cell disease and 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 doesn’t become sickle shaped but is usually turned off in infancy,” lead study investigator Vivien Sheehan, assistant professor of pediatrics at Baylor and Texas Children’s, said. “This is an exciting example of collaborative, bench-to-bedside research that has now resulted in a clinical trial that is already enrolling patients.”....