A cure for sickle cell disease may be in the making as Doctors in Paris have monitored and confirmed the success of a DNA reversal treatment carried out on a teenager 15 months ago.
Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with the disorder have defective haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle, or crescent, shape.
Doctors in Paris treated a teenager with the disorder by altering the genetic instructions in his bone marrow, so that it instead produced healthy red blood cells.
BBC reporter James Gallagher 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. After that, the teen underwent chemotherapy for four days to eliminate his diseased stem cells before the corrected bone marrow was replanted.
The BBC reports that Philippe Leboulch, a professor of medicine at the University of Paris said that following the procedure, “the patient has no sign of the disease, no pain, no hospitalisation … We are quite pleased with that.”
The condition of the teenager was said to be quite critical prior to the intervention by the Doctors. Accordingly, he had needed to undergo surgeries where his spleen was removed and his hips replaced. He also used to have a monthly blood transfusion to dilute his defective blood, something that he no longer requires.
Despite their laudable feat, Professor Leboulch is hesitant about asserting the treatment as a “cure” for sickle cell anaemia. It is, however, safe to say that the success of the pilot case is a significant milestone in the treatment of sickle cell disease.
Professor Leboulch said that 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.”
The celebration may also be dampened by the fact that this pioneering treatment is quite costly and can only be performed in highly advanced hospitals. For developing countries that may pose quite a challenge even if the treatment does gain mainstream status.