Duchenne Muscular Dystrophy (DMD) is a degenerative genetic disorder that affects the muscles in the body in a progressive manner, leading to eventual death. Mesenchymal stem cells (MSC) are able to reach the affected muscle cells and to start tissue regeneration and repair, making them an attractive treatment option for DMD. This study by Pelatti et al. demonstrates the safety of MSC to treat Golden Retriever Muscular Dystrophy – an animal disorder with remarkable similarities to human dystrophy. The treatment was well-tolerated and no long-term adverse effects were reported.
Stem Cell Rev. 2016 May 19. doi: 10.1007/s12015-016-9659-3
Transplantation of Human Adipose Mesenchymal Stem Cells in Non-Immunosuppressed GRMD Dogs is a Safe Procedure.
Abstract
The possibility to treat Duchenne muscular dystrophy (DMD), a lethal X-linked disorder, through cell therapy with mesenchymal stromal cells (MSCs) has been widely investigated in different animal models. However, some crucial questions need to be addressed before starting human therapeutic trials, particularly regarding its use for genetic disorders. How safe is the procedure? Are there any side effects following mesenchymal stem cell transplantation? To address these questions for DMD the best model is the golden retriever muscular dystrophy dog (GRMD), which is the closest model to the human condition displaying a much longer lifespan than other models. Here we report the follow-up of 5 GRMD dogs, which were repeatedly transplanted with human adipose-derived mesenchymal stromal cells (hASC), derived from different donors. Xenogeneic cell transplantation, which was done without immunosuppression, was well tolerated in all animals with no apparent long-term adverse effect. In the present study, we show that repeated heterologous stem-cell injection is a safe procedure, which is fundamental before starting human clinical trials.