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.
So far, the therapy has worked for 15 months and the child is no longer on any medication.
Doctors removed his bone marrow – the part of the body that makes blood. They then genetically altered it in a lab to compensate for the defect in his DNA that caused the disease.
The results in the New England Journal of Medicine showed the teenager has been making normal blood since the procedure 15 months ago.
Philippe Leboulch, a professor of medicine at the University of Paris, told the BBC: “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.
The study does show the potential power of gene therapy to transform the lives of people with sickle cell.
“I think it’s very significant, essential they’ve given him his life back,” said Dr Deborah Gill from the gene medicine research group at the University of Oxford.
She told the BBC: “I’ve worked in gene therapy for a long time and we make small steps and know there’s years more work. But here you have someone who has received gene therapy and has complete clinical remission – that’s a huge step forward.”
However, the expensive procedure can only be carried out in cutting-edge hospitals and laboratories, while most sickle cell patients are in Africa.
The next big challenge will be to transform this pioneering science into something that really can help millions of people.