How to prepare the health system at advanced therapies: this is the challenge taken up by a working group supported by Vertex Pharmaceuticals and made up of clinicians, patients, pharmaco-economists to guarantee rapid access to the most innovative medicines even for patients suffering from rare diseases or without therapeutic alternatives.
Advanced therapies represent an important development opportunity for Italian research and treatment for patients affected by rare diseases or without therapeutic alternatives. One of the most promising lines of research concerns the pathologies caused by the low production of hemoglobin in the blood. An Italian research team has developed a genetic manipulation therapy that could in the future allow people affected by beta-thalassemia no longer having to resort to transfusions.
Thalassemia: a future without transfusions
Today in fact thanks to the most advanced technologies it is possible to cure pathologies caused by the poor production of hemoglobin. Three years after the start of the trial, new data highlight the efficacy and safety profile of the first treatment to emerge from the research program, in patients with transfusion-dependent beta-thalassemia and sickle cell anaemia.
«The results demonstrate the positive impact that this therapy has had on patients and I cannot hide my satisfaction. Given the lack of definitively curative therapies for the majority of patients with hemoglobinopathies, I am thrilled to be part of the team that developed the “genome editing” approach with the potential to provide patients with a solution to this need dissatisfied therapeutic» he comments Frank LocatelliDirector of the Department of Onco-Hematology and Cell and Gene Therapy, IRCCS Pediatric Hospital Bambino Gesù in Rome.
The team coordinated by Professor Locatelli has developed a genetic manipulation therapy which will allow 90% of patients with beta-thalassemia to no longer have to resort to transfusions. An important perspective for the over seven thousand thalassemic patients living in Italy, for now only studied in young adults but which researchers will soon begin to test in children as well.