CHICAGO (Reuters) - Gene therapy has led to normal blood flow in children with thalassemia, according to a long-running international study. In this way, they no longer need frequent blood transfusions and are now living a normal life.
This important and international research has been going on for many years and its results have been published in the New England Journal of Medicine. After several years of gene therapy, 90% of the patients after the Phase 3 clinical trial still do not need fresh blood and are completely healthy.
All patients ranged in age from 4 to 34 years and all had undergone gene therapy. 90% of those under 12 years of age still did not need a blood transfusion on a monthly basis.
Dr. Jennifer Schneiderman, a specialist at the N&R Robert H. Lowry Children's Hospital in Chicago, is also part of the international research team. According to him, this research has been done for ten years in which transfusion thalassemia patients have been treated with gene therapy.
Hemoglobin in the blood of thalassemia patients slows down. In severe cases, the patient needs red blood cells every month. But repeated blood transfusions can lead to iron deficiency, infections and other painful conditions.
In this gene therapy, the patient's own stem cells were taken and taken to the laboratory. An altered variant was then added to replace the damaged gene that caused thalassemia. All patients then underwent some form of chemotherapy and had to undergo this procedure for four to five weeks. During this time, a team of doctors remained vigilant to deal with any disturbance. The stem cells were then inserted into the body.
Within a month, the majority of patients no longer needed repeated blood transfusions. But doctors repeatedly examined many patients for 13 months to four years and looked at the risks of thalassemia in the blood. The most severe effects of therapy were observed in four of these patients. Some people complained of blisters in the mouth, fever and other conditions, including a decrease in red and white blood cells.
But a total of 90% of patients with thalassemia disappeared and were freed from blood transfusions, including the majority of children.
