«I think there is no better way to celebrate this day. We have an extraordinary new therapeutic opportunity for thalassemia patients.” From Frankfurt, where he takes part in a conference, the enthusiasm of Franco Locatelli, oncohaematologist at the Bambino Gesù hospital, coordinator of the study on thalassemia, whose world day is celebrated today, reaches Sardinia. All the research data have just been published in the “New England Journal of medicine”.
For the island, where the pathology affects a thousand people (out of a total of 7000 thalassemia patients in Italy), the turning point that many patients have been waiting for. «The study demonstrated that through genetic editing, based on very selective intervention, a sort of molecular scissors on a particular gene called Bcl11a, there is the possibility of reactivating fetal hemoglobin».
This year’s World Thalassemia Day brings a wind of renewed hope and confidence in the future to the Microcitemico hospital in Cagliari, where the Microcytemia and Rare Anemia Center directed by Susanna Barella operates, which follows 496 patients for therapies and transfusions (half of Sardinian thalassemia patients). «Three patients from our Centre, two teenagers and a thirty-five-year-old – he explains – underwent the experimentation conducted by Professor Locatelli. For three years they have been totally independent of transfusions. The new therapeutic strategy will have a very strong impact on the treatment of thalassemia because it is the first treatment that can lead to recovery without the need for a compatible donor. It is the objective that all specialists involved in the treatment of thalassemia have always tried to achieve. I think that between the end of this year and the first months of 2025 it will be possible to use gene therapy in our centers.”
In the video the interviews with Franco Locatelli and Susanna Barella.
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