A French teenager's sickle cell disease has been reversed using a pioneering treatment to change his DNA. The world-first procedure at Necker Children's Hospital in Paris offers hope to millions of people with the blood disorder. Scientists altered the genetic instructions in his bone marrow so it made healthy red blood cells.
The teenager was prior to the treatment on a monthly blood transfusion to dilute his defective blood. At age 13, doctors at the Hospital in Paris decided to alter the genetic instructions in his bone marrow.
This was done through the injection of a virus (which corrected the defect he had) into his bone marrow. The altered bone marrow was then put back into the patient.
Fifteen months since the treatment, the teenager no longer requires medication, and his blood cells show no further sign of the disease, according to the results published in the New England Journal of Medicine.
Philippe Leboulch, a professor of medicine at the University of Paris, told the BBC that: “So far the patient has no sign of the disease, no pain, no hospitalisation. He no longer requires a
transfusion so we are quite pleased with that.
“But of course we need to perform the same therapy in many patients to feel confident that it is robust enough to propose it as a mainstream therapy.”
Leboulch, according to the BBC, is hesitant to use the word “cure” because the French teenager is only the first patient to scale through the clinical trials.