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Shown to Ease Muscular Dystrophy in Dogs
Canada AM
17 October 2006
Host(s): Seamus O'Regan
Guest(s): Dr. Robert Worton, Sprott Centre for Stem Cell Research
O'REGAN: There is promising new research out of Italy where stem cells have worked well at easing symptoms of muscular dystrophy in a group of golden retrievers, a result that experts call a significant step toward treating people. To talk about the new findings, Dr. Robert Worton of the Sprott Centre for Stem Cell Research joins us from Ottawa.
Doctor, thanks very much.
SPROTT: Good morning.
O'REGAN: Okay, so far we've been able to ease the plight of dogs with muscular dystrophy. Tell me about the significance of this for humans.
SPROTT: Well, the significance for humans is that these dogs represent an absolutely true model of Duchenne muscular dystrophy in humans. Duchenne muscular dystrophy is one of the more severe forms of muscular dystrophy. It affects young boys, usually at around age four or five. And it's a quite devastating disease. They're usually into a wheelchair by about age six, or at the latest nine. The problem with Duchenne muscular dystrophy is that the kids are missing a protein called dystrophin in their muscle. And that results in muscle degeneration over the course of time. So, we've known for a long time that the only way we can really help these kids is to rebuild their muscle after it has degenerated. And we've always thought that stem cells might be the answer. So, this study, by Giulio Cossu from Italy, is really the first in dogs to show a dramatic effect. And so, that's the real significance for humans.
O'REGAN: Bridge this gap for me, though. Stem Cell 101, if you would. What happens? The stem cells determine that the body needs new muscles, and then immediately morph into those muscles or trigger the growth of those muscles? Is that how it works?
SPROTT: In a way that's true. Our stem cells are cells that haven't yet specialized into anything in particular. And the stem cells that they used were actually taken from muscle tissue. And part of their job is to make muscle in the normal animal. So, what they did was in the best experiments is they injected about 50 million of these stem cells from muscle into the aorta, just at the output of the heart, so that the bloodstream then would carry them throughout the body. And these stem cells are able to cross out of the bloodstream and into the muscle. And once they were into the muscle then, of course, they were in an environment where they are stimulated to make new muscle tissue.
O'REGAN: How interesting. When do you think something similar could be used for human beings?
SPROTT: Yeah, that's a difficult question, and always is. But I can tell you that I'm optimistic now for the first time in a long time. I think these are proof-of-principle studies. Not all of the dogs got better. Of the six dogs that they did with the optimal procedure, two actually died, two were stabilized and didn't get worse, and two actually improved. One of them was actually walking quite well after five months when he hadn't been able to walk very well before. So, two dogs getting better out of six, that's not enough to be able to start doing research in humans. But it's enough to tell us that with some tweaking of the system and some additional experiments in dogs, perhaps over the next year or two, it may work much better. And as soon as we get to that point there will be a number of groups, including groups in Canada, who will be ready to try this in humans.
O'REGAN: As soon as you get to that point you let us know.
SPROTT: We will do that, for sure.
O'REGAN: That is absolutely fantastic. Thank you, sir, very much.
SPROTT: You're very welcome.
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