ALLOTRANSPLANTATION OF MESOANGIOBLASTS IN DUCHENNE MUSCULAR DYSTROPHY: A PHASE I CLINICAL TRIAL

Cell therapy is one promising approach to correct genetic diseases by contributing to tissue regeneration; stem cells can be isolated from a healthy donor or, when possible from the same patient. In the first case cells will be transplanted under a regime of immune suppression while in the second case, cells will have to be genetically corrected before transplantation in the same patient from which they were derived. The recent identification of different types of stem cells, some of which are suitable for protocols of cell therapy, has disclosed new perspectives in the treatment of genetic diseases. Our previous work indicated that a recently identified population of stem cells - the mesoangioblasts - produce functional improvement upon intra-arterial injection in mouse and dog models of muscular dystrophy. Moreover the corresponding human cell have been characterized. Based on these results we propose a pilot clinical trial, based on intra-arterial transplantation of normal mesoangioblasts under a regime of immune suppression. Efficacy and possible adverse effects and will be evaluated to test whether this approach may represent a first step towards an efficacious therapy for muscular dystrophy.