Beta-thalassemia is an inherited disorder in which the body is unable to make normal hemoglobin and red blood cells. Hemoglobin is the protein in red blood cells that carries oxygen. Patients typically need repeated transfusions during their lifetimes, but this treatment is not a cure for the disease.
In this study, researchers are evaluating a new method for treating beta-thalassemia which relies on an approach called “gene transfer.” They will collect from the blood some of a patient’s own stem cells (blood-forming cells made in the bone marrow). The cells are then treated so they contain the corrected gene for making normal hemoglobin. The treated cells are returned to the patient through an intravenous infusion (by vein), and the patient also receives a drug called busulfan — before the infusion — to prepare the body to receive the new stem cells.
The goal of this phase I study is to assess the safety of this approach and to see if the treated stem cells with the new gene make their way back to the bone marrow without causing side effects. The hope is that this treatment can make a patient’s body produce normal hemoglobin and red blood cells.