French Doctors Invent New Treatment for Sickle Cell Disease

A huge innovation was made at Necker Children’s Hospital in Paris, France — the world’s first successful treatment for sickle cell disease (SCD). A French teenager with sickle cell disease, who had gone through working therapy for fifteen months, does not have to use medications any longer. The French teenager reported that he no longer had to deal with the pain from the disease. Doctors were able to modify the teenager’s bone marrow genetics so the damaged red blood cells could be healthy.

What is sickle cell disease?

• Sickle cell disease is a group of disorders that cause red blood cells to become deformed or break down.
• The symptoms include, but are not limited to: fatigue, infections and pain
• Some forms of sickle cell disease are Hemoglobin SS, Hemoglobin SC Hemoglobin Sβ+ thalassemia, Hemoglobin Sβ0 thalassemia, Hemoglobin SD and Hemoglobin SE.
• In contrast to normal hemoglobin in cells, sickle hemoglobin can form stiff rods changing the cell into a crescent shape.
• When the cells affected by sickle hemoglobin become inflexible, they stick to vessel walls, blocking the flow of oxygen in blood.

Before the teenager had his special treatment, he had to go to the hospital monthly for blood transfusion for his unhealthy blood. When he was thirteen, the hospital started with their new treatment.

The doctors removed the boy’s bone marrow. Then, they genetically altered the extracted marrow in a lab to fix the problems in his DNA that had given him the sickle cell disease. The French doctors inserted a virus to correct the malfunctioned DNA, and soon after, they put the marrow back into the teenager’s body.

Since a year and two months ago, the patient has been making normal, healthy blood, according to the New England Journal of Medicine.

Prof. Leboulch expressed his worries over the new treatment being branded as a “cure”, because only one patient had went through the process of the treatment. Unfortunately, many people would currently be unable to receive the same treatment because of the lack of funds for it in their countries. Hopefully, in the future, anyone who is suffering from the disease will be able to get full treatment.