According to the National Heart, Lung and Blood Institute, stem cell transplantation (HSCT) is the only cure for sickle cell disease. You read that correctly. Yes, there is a cure for sickle cell disease. However, it hasn’t been an easy cure. Stem cells are precursors to fully formed cells, meaning that when infused into the body, they eventually grow into the desired cells. In this example, healthy, blood-forming stem cells are given to replace the diseased sickle cells. Treatment challenges have included the need to find a suitable donor and the need to undergo chemotherapy prior to the transplantation. Until recently, HSCT was only being performed at the National Institutes of Health in Maryland, where a success rate of 87% has been reported in the treatment of 30 patients. More recently, physicians at the University of Illinois have picked up the mantle. They have recently reported 12 cures in the treatment of 13 adult sickle cell patients, representing a 92% success rate.
The success of the University of Illinois is largely attributable to an alteration in the procedure that has eliminated the need to undergo chemotherapy. The significance of that last sentence is substantial. The illness itself often renders patients too ill to endure the risks of chemotherapy. These risks include killing off the patient’s own blood-forming cells and virtually freezing their immune systems as a means of avoiding rejection of the transplanted cells by the body’s active immune system. As a reminder, a major risk of immunosuppression is dangerous, life-threatening infection. Not having to address that risk will introduce the opportunity for a cure for tens of thousands of patients.
This is an important application of stem cell therapy. Treatment of sickle cell patients with HSCT produces enough healthy red blood cells to eliminate symptoms and even detection of sickle cells.
If you, someone in your family or a friend has sickle cell disease, please discuss this procedure with your physician. Having a cure for a disease is only as good as your awareness of it and ability to take advantage of it.
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