Treatment Options For Thalassemia

Stem Cell Transplants

Thalassemia can be treated with blood transfusions, but it can only be cured by stem cell transplants. All human cells arise from stem cells. Stem cells are primordial, able to become many different kinds of cells as the fetus grows. There are two types of stem cells: adult and embryonic. Adult stem cells can be obtained from bone marrow and from the blood in umbilical cords, which is routinely collected and banked in many places now. Embryonic stem cells are obtained from embryos, usually those leftover from procedures done at fertility clinics.

Obtaining adult stem cells generally harms no one, though obtaining embryonic stem cells destroys the embryo, which leads to ethical questions. Stem cell transplants are complex and fraught with dangers to the patient. The patient's bone marrow, which produces abnormal cells, must be destroyed and then replaced with the stem cells. Infection and host versus graft disease are the major concerns, both of which can be fatal. However the chances for a cure for thalassemia, even in adults, is usually far greater than the risks of the procedure. Cure rates are much higher in children with thalassemia than in adults (sixty-five percent) who have more the advanced disease.