A ray of hope is now in view for sickle cell patients all over the world as a scholastic group of French doctors have successfully developed a cure for sickle cell, a disease that has plagued many families for years.
According to BBC News, the discovery was confirmed a success after a French teenager’s sickle cell disease was reversed using a pioneering treatment to alter his DNA.
The procedure which is the world’s first was carried out at Necker Children’s Hospital and offers hope to millions of people with the blood disorder. The report revealed that alterations were made on the genetic instructions of a teenager in his bone marrow which made healthy red blood cells. The therapy has worked for 15 months so far and the child is no longer on any medication.
The next big challenge will be to transform this pioneering science into something that really can help millions of people.
The teenager who received the treatment had so much internal damage he needed to have his spleen removed and his hips replaced.
He had to go into hospital every month to have a blood transfusion in order to dilute his defective blood.
But when he was 13, something unique was done by doctors at the Necker Children’s Hospital in Paris.
His bone marrow – the part of the body that makes blood was removed. They then genetically altered it in a lab to compensate for the defect in his DNA that caused the disease. A virus was used to infect the bone marrow with new, correct instructions. The corrected bone marrow was then put back into the patient.
Ever since this procedure, the teenager has been making normal blood for the past 15 months.
However, Philippe Leboulch, a professor of medicine at the University of Paris, is nervous about using the word “cure” as this is just the first patient to come through clinical trials.
But the study does show the potential power of gene therapy to transform the lives of people with sickle cell.
Sickle cell is a disease which causes normally round red blood cells, which carries oxygen around the body, to become shaped like a sickle. These deformed cells can lock together to obstruct the flow of blood around the body, leading to intense pain or organ damage and can be fatal in some cases.