Protein injection holds promise for muscular dystrophy treatment
Injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, scientists have discovered.
The findings could lead to a therapy akin to the use of insulin by diabetics.
The study was conducted by Dr. Julia von Maltzahn and Dr. Michael Rudnicki, the Ottawa scientist who discovered muscle stem cells in adults.
"This is an unprecedented and dramatic restoration in muscle strength," said Dr. Rudnicki, a senior scientist and director for the Regenerative Medicine Program and Sprott Centre for Stem Cell Research at the Ottawa Hospital Research Institute.
He is also a Canada Research Chair in Molecular Genetics and professor in the Faculty of Medicine at the University of Ottawa.
"We know from our previous work that this protein, called Wnt7a, promotes the growth and repair of healthy muscle tissue. In this study we show the same types of improvement in a mouse model of Duchenne muscular dystrophy. We found that Wnt7a injections increased muscle strength almost two-fold, to nearly normal levels. We also found that the size of the muscle fibre increased and there was less muscle damage, compared to mice not given Wnt7a," he explained.
Duchenne muscular dystrophy is a genetic disorder that affects one of every 3,500 newborn males. In Canada, all types of muscular dystrophy affect more than 50,000 people. The disease often progresses to a state where the muscles are so depleted that the person dies due to an inability to breath. For people with Duchenne muscular dystrophy, this usually happens in their 20s or 30s.
"This is also exciting because we think it's a therapeutic approach that could apply to other muscle-wasting diseases," said Dr. Rudnicki.
These results were published in the Proceedings of the National Academy of Sciences.