Gene therapy of hereditary diseases: another success
Gene therapy helped a Frenchman with a serious illness
Sofya Lopaeva, VademecumA thirteen-year-old Frenchman suffering from sickle cell anemia was able to do without blood transfusion for three months after gene therapy.These are the data of Bluebird Bio clinical trials presented at the meeting of the European Society of Hematology (EHA) in Vienna.
A French young man has become the first sickle cell anemia patient to be treated with Bluebird Bio's LentiGlobin BB305 technology, according to Reuters (Bluebird Bio's sickle cell gene therapy working for French boy). Sickle cell anemia is a hereditary blood disease characterized by deformation of red blood cells: instead of an even, ring-shaped shape, they take a sickle–shaped one, losing flexibility and elasticity. Deformed red blood cells can clog blood vessels, disrupt blood flow, lead to anemia and, in some cases, premature death. The cause of sickle cell anemia is a mutation in the hemoglobin gene.
Blubird Bio's gene therapy involves extracting blood stem cells and replacing the mutant gene in them with its working copy. After this procedure, stem cells capable of turning into healthy red blood cells are injected into the patient. After participating in clinical trials of this technique, the French patient was able to do without blood transfusion for three months, and the hemoglobin level in his blood increased significantly.
Other participants in clinical trials – two patients suffering from a related disease – beta-thalassemia, were able to refuse blood transfusion for 16 and 14 months. Bluebird Bio has previously reported a shorter effect of its gene therapy for people with beta thalassemia.
Bluebird Bio's shares have doubled this year amid reports of good clinical trial results, and the company's CEO Nick Leschley was included in the list of the most influential persons in the biotechnology industry. According to a number of experts, in order to understand the potential of gene therapy developed by Bluebird Bio, data on its effectiveness obtained with a larger number of patients is needed.
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17.06.2015