Success in Gene Therapy of Hemoglobin Disorders

European school of hematology reports first success in gene therapy of hemoglobin disorders. Twenty years after the first transplantation of cord blood cells, a new milestone in biotherapies has been reached by the first gene therapy trial for β-thalassaemia, performed in Paris. The challenge was to treat adult thalassaemia major patients who did not have an HLA matched donor.
Тhe results are encouraging. They show that a 21 year old patient with severe β-thalassaemia, who was previously under a life long transfusion programme has been free of all transfusions 1 year after the transplantation of his corrected bone marrow cells. The therapeutic haemoglobin remains stably expressed (3.5 g/dL of blood) raising the Hb blood level to 9.5-10 g/dL. The patient feels cured and says that he has new strength, a new life and a full time job. Iron overload is slowly removed by oral chelation. It appears probable that the patient will be able to stop chelation in the near future. This preliminary result needs to be confirmed by inclusion in the trial of other patients with severe thalassaemia or sickle cell anaemia.
Many other genetic or acquired disorders could be future candidates for this type of gene therapy treatment. To read the full article, please click here.