Preclinical experience and perspectives of a clinical trial using CD133 stem cells

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 dystrophic patient. In the first case cells will be transplanted under a regime of immune suppression; in the second case, cells will have to be genetically corrected before transplantation in the same patient. The recent identification of different types of multi-potent stem cells, some suitable for protocols of cell therapy, has disclosed new perspectives in the treatment of genetic diseases. Our previous work indicated that CD133+ cells, a population of progenitor cells, produce functional improvement upon intra-arterial injection in a dystrophic mouse. Transplantation of engineered dystrophic canine muscle-derived CD133+ cells gave promising results in GRMD dogs, the most reliable animal model that shows a clinical phenotype very similar to DMD patients. Because of these results, we plan a pilot clinical trial, based on intra-muscular and intra-arterial transplantation of autologous engineered CD133+ cells. Efficacy and possible adverse effects will be evaluated to test whether this approach may represent a first step towards a therapy for muscular dystrophy.