Thanks to the constant advancement of Science, a cure may have been discovered for sickle cell disease. Doctors in Paris have monitored and confirmed the success of a DNA reversal treatment carried out on a teenager 15 months ago. Since the disease is such that the bone marrow produces deformed red blood cells, scientists altered the genetic instructions in his bone marrow, so it produced healthy red blood cells.
James Gallagher for BBC reports that the teenager had his bone marrow taken out, stem cells harvested and genetically altered with a virus to infect it with correct instructions. Next, he underwent chemotherapy for four days to eliminate his diseased stem cells, before the corrected bone marrow was replanted. Since then, the teenager has received a clean bill of health. “So far the patient has no sign of the disease, no pain, no hospitalisation … We are quite pleased with that,” Philippe Leboulch, a professor of medicine at the University of Paris, told the BBC.
Prior to the surgery, the condition of the teenager is said to have been critical. So much that he needed to have his spleen removed and his hips replaced. He also used to have a monthly blood transfusion to dilute his defective blood. But now, he no longer requires a transfusion.
Although Professor Leboulch is hesitant about asserting the treatment as a “cure” for sickle cell anaemia, the success of the pilot case is a significant milestone in the treatment of the disease. According to the professor, there needs to be more performance of this therapy on several patients to create a certainty that it is “robust enough to propose it as a mainstream therapy.”
However, just as the bone marrow transplant, the only known cure for sickle cell anaemia is expensive, this pioneering treatment is quite costly and can only be performed in highly advanced hospitals. This already poses a huge challenge to the African continent where the disease is predominant, as the cost implications are bound to widen between developed and developing countries, largely due to exchange rates.