Dogs with Duchenne muscular dystrophy experienced a return of muscle strength after receiving stem cell therapy, say scientists from Italy and France. Duchenne muscular dystrophy affects about one in every 3,500 male humans – a genetic mutation means a gene lacks dystrophin, a crucial protein for muscle health. The patient’s muscles gradually weaken, he eventually loses mobility and usually dies young.
You can read about this study in the journal Nature.
The scientists believe this study could eventually lead the way to effective treatment for humans with muscular dystrophy (MD).
Previous studies on mice by the same team had indicated that stem cell therapy had promise for MD. They decided to carry out a further study on dogs, as dogs suffer from MD in a more similar way to humans (compared to mice).
The scientists gathered mesoangioblasts – stem cells which turn into muscle cells. Mesoangioblasts were collected from healthy dogs and dogs with MD. The mesoangioblasts from the MD dogs had their genes modified so that the mutated gene was corrected. They then injected mesoangioblasts into the MD dogs once a month for five months.
They found that the MD dogs which received mesoangioblasts from healthy dogs benefited more than those which received ‘corrected’ mesoangioblasts.
Of six dogs with MD, four began to experienced a return of dystrophin. The same four dogs also regained muscle strength. One of the dogs, which was treated early on during his MD, did not lose his ability to walk. Another two, which had more advanced MD, managed to get back their mobility (they could move again). Two dogs did not benefit – one died while the other experienced no improvement. The scientists believe the two dogs which did not benefit should have been given higher doses.
When humans with MD are eventually treated with stem cell therapy for MD, it would be ideal to use the patient’s own corrected stem cells. However, the scientists found that the dogs which received stem cells from healthy dogs did much better than those which received their own corrected cells. It is hoped that further research will overcome this difference. If a patient can receive his own cells he will not need to take immunosuppressant drugs (due to rejection).
Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
Maurilio Sampaolesi, Stephane Blot, Giuseppe D’Antona, Nicolas Granger, Rossana Tonlorenzi, Anna Innocenzi, Paolo Mognol, Jean-Lauren Thibaud, Beatriz G. Galvez, Ines Barth?l?my, Laura Perani, Sara Mantero, Maria Guttinger, Orietta Pansarasa, Chiara Rinaldi, M. Gabriella Cusella De Angelis, Yvan Torrente, Claudio Bordignon, Roberto Bottinelli and Giulio Cossu
Nature doi:10.1038/nature05282
Written by: Christian Nordqvist
Editor: Medical News Today