Muscular Dystrophy: Stem Cell Help?
Stem Cell Treatment Shows Potential in Lab Tests on Dogs
Nov. 15, 2006 -- Lab tests on dogs show that adult stem cells may help treat
a certain type of muscular dystrophy, say Italian researchers in the journal
They included Giulio Cossu, MD, of the Stem Cell Research Institute in
The strategy needs more work. But Cossu's team says their study "sets
the logical premise for the start of clinical experimentation" for Duchenne
Duchenne muscular dystrophy is one of the nine major types of muscular
dystrophy. It's the most common form of muscular dystrophy in children, and it
only affects males.
In human Duchenne muscular dystrophy, muscles weaken over time. Many
patients eventually need wheelchairs; most die in their late teens or early
Stem Cell Study
Cossu and colleagues studied 13 male golden retrievers that had a canine
form of Duchenne muscular dystrophy.
The researchers took adult stem cells from the dogs with muscular dystrophy
and from a dog without muscular dystrophy.
Cossu's team split the dogs with muscular dystrophy into three groups.
The first group included three dogs that got no stem cell shots. Their
muscular dystrophy worsened.
The second group included six dogs that got five shots of donated stem cells
from the healthy dog, along with drugs to help their immune systems accept
The last group included four dogs that got up to five shots of their own
stem cells that had been tweaked to make more dystrophin, a protein lacking in
Duchenne muscular dystrophy.
The results varied, but the dogs that got the donated stem cells generally
Two of the six dogs that got donated stem cells were able to walk, one of
which was able to run at the end of treatment.
A third dog in the same group died suddenly, and a fourth gradually lost
walking ability after drugs for the immune system were stopped.
The fifth dog that got donated stem cells showed no improvements or
setbacks, and the sixth showed "modest" worsening of its condition.
Of the four dogs that got shots of their own genetically tweaked stem cells,
one was able to walk stiffly after treatment. Three worsened, two of which died
Muscular dystrophy worsened in all of the dogs that didn't get stem cell
The donated stem cells "seemed to be more efficient" than the
genetically tweaked stem cells, the researchers write.
But transplanting stem cells can have drawbacks, notes editorialist Jeffery
Chamberlain, PhD, of the University of Washington's neurology department.
"As with any transplant, unless the donor and recipient are
immunologically matched, the recipient must be placed on lifelong immune
suppression; this is not always effective and can have nasty side effects,"
Chamberlain writes in Nature.
He adds that it would be "preferable" to give each patient their own
genetically corrected stem cells, if the technique can be made more