Sardinian thalassemic patients moving toward a ban on transfusions: new gene therapy presented at Brotzu Hospital
On January 13th, the blood sample was collected from "Patient 0." The cells were processed in a U.S. laboratory and an accredited center in Cagliari.Video di Egidiangela Sechi
Sardinian patients with thalassemia are increasingly closer to eliminating periodic transfusions thanks to gene therapy for beta-thalassemia . The turning point is set for January 13th, when the first Sardinian patient with the disease will undergo a hematopoietic stem cell harvest at Arnas Brotzu in Cagliari . The cells, once harvested, will be sent to the United States and returned to Cagliari with the "reprocessing" that will allow the body to produce functional hemoglobin. Once reinfused into the patient, they will pave the way for a possible life without transfusions.
Brotzu, the only accredited center in Sardinia , is thus launching the first advanced procedure of its kind in Italy, marking a historic step in the treatment of beta-thalassemia . There are approximately one thousand patients in Sardinia: the treatment is intended for clinically eligible patients between the ages of 12 and 35. "Our region has always experienced a high incidence of thalassemia, a disease that has profoundly affected the lives of many families," explained Arnas Brotzu Special Commissioner Maurizio Marcias . "We are proud to be able to say that we are among the first companies in Italy to perform the treatment among those that did not participate in the trial. We will continue to work to ensure that this innovation becomes fully accessible to the many Sardinians affected by thalassemia, with maximum safety and with the support of the best clinical expertise available."
By January, two patients will undergo the new therapy . The treatment involves harvesting stem cells from the patient's peripheral blood, genetically modifying them in a US laboratory—using gene-editing techniques that reactivate fetal hemoglobin production—and finally reinfusion via autotransplantation of genetically modified hematopoietic cells. "Thalassemia is a quality-of-life issue," noted Medical Director Giorgio La Nasa , "because thalassemia patients require periodic transfusions, even very frequently, sometimes twice a month. With this genetic remodeling system, the patient becomes independent. Forever."
(Unioneonline)