Gene therapy, a revolutionary method that has been proving useful for the treatment and even cure of many hard-to-cure diseases, has made yet another triumph for mankind, this time for sickle cell disease. Though doctors are still hesitant to call the therapy a cure for sickle cell disease, it is a major step toward combating the disease.
A study published in The New England Journal of Medicine on March 2 sheds light on the case of a French teenager with sickle cell disease and the gene editing therapy that could possibly have completely reversed the patient's disease.
Life Changing Treatment?
The boy began the treatment 15 months ago when he was brought to the Necker Children's Hospital in Paris in 2014. What his doctors did was alter the code in his DNA by adding a specific "anti-sickling" gene to his stem cells.
Once introduced into the body, the hope was that the newly altered genes would then translate into the production of healthy red blood cells instead of sickly sickle cells that cause a number of bodily complications.
The study stated 15 months after the very first treatment that the boy who once had significant internal damage — so much so that it led to a removed spleen and a hip replacement — now has no sign of the disease. He is no longer experiencing any of the usual markers of sickle cell disease and, at the least, 50 percent of his blood cells are considered normal and healthy. The boy is also no longer under any medication and treatment related to the disease.
Six other patients were also treated with the disease, though it seems the treatment worked best for the teenage boy.
Sickle Cell Disease
Sickle cell disease is an inherited blood disease that affects hemoglobin, the component of red blood cells responsible for delivering oxygen to other cells all over the body. Individuals with sickle cell disease have red blood cells that are sickle or crescent-shaped instead of the usual, healthy round shape. Characteristics of the disease include anemia, frequent infections, and episodes of pain.
Though not all patients experience serious symptoms, others require frequent hospitalization and experience serious and severe symptoms.
There is no current cure for sickle cell disease, but scientists are continuing in their search for therapies and treatments, such as bone marrow transplants and blood transfusions, which could hopefully alleviate the disease in the future altogether.
