OA fee for Innovator Award
Wellcome Trust
Rare Partners (Italy)
The availability of funding will allow completion of a first exploratory clinical trial on sirolimus. This would be the first trial with the drug in thalassemia, will involve about 20 patients and will explore a surrogate end point known as an important point in thalassemia, namely HbF. The trial will also explore other end points, trying to evaluate meaningful clinical end points at a preliminary stage. The final goal of the project is a substantial decrease of the transfusion need, of great interest for the thalassemia patients. A reduction of transfusion need would dramatically improve the quality of life and reduce the iron overload, caused by repeated transfusions of red blood cells. The strategy for the future clinical development of sirolimus in thalassemia will be designed taking into account the results of this first pivotal trial. An additional important output of the study of particular significance would be the advancement toward personalised medicine (ex vivo test to predict in vivo results) and the possible combination of agents acting on HbF to improve treatment approaches. The available data, obtained so far in vitro on human preparations, strongly support this view and would be confirmed and expanded by the planned experiments.
5.2 Cellular and gene therapies