Tgcom24
The “ultimately curative potential” of the strategy
The studies were published in the “New England Journal of Medicine” and confirm “the definitive curative potential” of this therapeutic strategy, said Franco Locatelli, head of the clinical and research area of Oncohematology of Bambino Gesù in Rome, who coordinated one of the two studios and is the second signature of the other.
The two most frequent hereditary blood diseases in the world
Thalassemia and sickle cell anemia are the two most frequent hereditary blood diseases in the world. Both pathologies are caused by mutations in the genes involved in the synthesis of hemoglobin, the protein in red blood cells that transports oxygen in the body.
Modification of hematopoietic stem cells
The two trials, promoted by the pharmaceutical companies Vertex Pharmaceuticals and Crispr Therapeutics, verified the therapeutic efficacy of an approach that involves the modification of hematopoietic stem cells through the CRISPR-Cas9 “molecular scissors” system. The modification serves to make the blood cells produce fetal hemoglobin instead of what is physiologically produced after birth.
91% achieved transfusion independence
The first of the trials involved 52 patients between 18 and 35 years old with beta thalassemia, of which 14 enrolled at Bambino Gesù and showed that 91% achieved independence from transfusion, obtaining on average hemoglobin values higher than those observed in parents, who are healthy carriers of the pathology. The high hemoglobin values and the presence of modified cells, in some of them, have persisted for over 4 years.
The therapy that frees you from vaso-occlusive crises
The second study instead confirmed, in 44 patients with sickle cell anemia (7 of which were treated at Bambino Gesù), that the therapy is able to free them from vaso-occlusive crises for at least 12 months. Also in this case the hemoglobin levels are good and the benefit is sustained over time.
Therapy approved by the EMA for patients over 12 years of age
The genome editing therapy, whose name is Exagamglogene Autotemcel, was approved a few months ago by the European Medicines Agency (EMA) for patients over the age of 12. Trials are underway on younger children. Baby Jesus is also in this research and has already treated 4 children with encouraging results.
“A milestone for the change of therapeutic scenario”
“The joint publication of the two studies in a journal such as the “New England Journal of Medicine” represents a milestone for what is the change in therapeutic scenario”, says Locatelli. “A result that demonstrates once again the ability and determination of the Bambino Gesù Pediatric Hospital to invest in innovative therapies capable of changing the natural history of such complex diseases”, he concludes.
