Talking about healing for patients with beta thalassemia is no longer taboo. Franco Locatelli, director of the department of Onco-Hematology and Cell and Gene Therapy of the Bambino Gesù Hospital in Rome, does so, commenting to ANSA on the results of a clinical trial published in the New England Journal of Medicine. The study, of which Locatelli is the first signatory, showed that 90% of patients treated with a gene therapy approach do not need transfusions.
Beta thalassemia is due to mutations in the HBB gene that can cause reduced or absent synthesis of hemoglobin beta chains. This causes life-threatening anemia that must be treated with regular transfusions. The gene therapy under study is called Betibeglogene autotemcel (beti-cel) and corrects this defect in the patient’s hematopoietic stem cells by adding functioning copies of the gene.
The trial, which started in 2016, was conducted in nine centers in Italy, France, Germany, Thailand, the United Kingdom and the USA and involved 23 patients with transfusion-dependent beta thalassemia: 8 children under the age of 12 and 15 people aged 12 and over. at the age of 50. The Infant Jesus contributed significantly, treating one third of the enrolled patients.
The results of the trial showed that the treatment “is able to determine transfusion independence in 90% of treated subjects. It was also able to determine the achievement of very consistent hemoglobin values in a high percentage of patients who obtained transfusion independence. This result is persistent over time “, says Locatelli. “When you have such important follow-up data, you can talk about healing,” he adds.
Currently, autotemcel betibeglogene therapy is approved by the EMA for people aged 12 and over with a specific genetic trait (non-beta0 / beta0 genotype) who need transfusions and do not have conditions incompatible with transplantation (for example, heart or liver problems ). However, the study also showed excellent results in children under the age of 12: “it is therefore foreseeable that the regulatory agencies will extend the indications to even the youngest children”, Locatelli affirms. Access to the drug, however, is currently complicated by disagreements over the reimbursement price between the company that developed the product and European drug agencies.
Gene therapy, however, is not the only advanced therapy potentially resolving beta thalassemia: “We have developed an approach based on genome editing, through which the synthesis of fetal hemoglobin is reactivated. With this strategy, too, we have achieved very important results. : all treated patients stopped receiving transfusion support “, concludes Locatelli.
Source: Ansa
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