The new drug luspatercept, now under development in the United States, could help to significantly reduce or even eliminate the need for transfusions for thalassemia patients, the medical consultant of the Thalassemia International Federation Michalis Angastiniotis told the Athens-Macedonian News Agency (ANA) on Monday.
Speaking on the sidelines of a Balkans Thalassemia Conference taking place in Thessaloniki, Angastiniotis said that clinical trials were now in their third phase and are expected to be completed in roughly 18 months.
He said that luspatercept acts on the red blood cells during their formation, increasing the number of healthy red blood cells in the circulatory system. In mild cases of thalassemia, where a patient was not fully dependent on transfusions, it could eliminate the need for them entirely. In more severe cases, where a patient was fully dependent, the number of tranfusions necessary are reduced by 40 pct on average but this can rise to as much as 80 pct for some individuals.
Fewer transfusions would mean fewer complications, thus also reducing the necessity for the close monitoring that thalassemia patients normally need and greatly improve their quality of life, he added.
Angastiniotis noted that gene therapy was also entering the third phase of clinical trials at this time, with 25 patients having undergone treatment throughout the world. The first patient that started the treatment seven years ago did not need transfusions but also suffered some negative side-effects, which Angastiniotis said was “not strange, given that it is the first time a treatment of this kind was administered.”
More recent cases had given “relatively good results,” he said and there were signs of further improvements in the future.
Thessaloniki will be among the first cities in the world where trials for both luspatercept and gene therapy for thalassemia will take place, at the Papanicolaou Hospital’s Gene and Cell Therapy Centre, which is among the most advanced in Europe.