STEM CELL RESEARCH
EuroStemCell one step closer to helping MD sufferers walk again
As stem cell research grabs headlines as a hot button issue,
concrete scientific results can sometimes get lost in the
fray. EuroStemCell, the European Consortium for Stem Cell
Research, has been making significant advances in laboratories
across Europe finding innovative solutions to a myriad of
diseases. Most recently they have produced promising results
for treating the debilitating, often fatal, genetic disorder
muscular dystrophy. They published their findings in a recent
issue of Nature.
EuroStemCell researchers have been able to temporarily restore
a dog’s ability walk after the advanced stages of muscular
dystrophy have set in. The prospects of such treatment have
enormous implications for humans suffering from the disease,
and researchers hope to move on to clinical trials for people
as early as next year.
deterioration caused by Duchenne MD significantly
reduces a patient’s life expectancy.
Muscular dystrophy is a general term referring to a range of
genetic disorders that affect muscle cells. It is characterised
by progressive muscle weakness and death of muscle cells and
tissue. Symptoms can vary widely with some patients experiencing
limited mild effects over a normal life span to others suffering
from severe disabilities. In some cases it can lead to death.
There is currently no cure, making EuroStemCell’s research
all the more exciting.
EuroStemCell researchers from the San Raffaele Scientific Institute
in Milan, Italy, led by Dr Giulio Cossu, have been studying
the most common form of the disease, known as Duchenne muscular
dystrophy. They discovered that the mesoangioblast muscle stem
cell, present in the walls of blood vessels, produced encouraging
results when injected in mice.
They decided to then test their findings on golden retrievers
carrying a mutation of the dystrophin gene, which closely resembles
the root of the disease in humans. The team took mesoangioblast
stem cells from the blood vessels of dogs with the mutation,
corrected it using gene therapy, and re-injected the modified
stem cells. They also repeated the procedure with cells from
healthy dogs, using drugs to prevent immune rejection.
Both treatment procedures resulted in the increased production
of dystrophin, though the injection of the donor stem cells
yielded the most encouraging results. Repeated doses of cells
from the healthy dogs restored muscle function in four of five
dystrophic dogs. All four lived for more than a year after the
experiment, and initially regained the ability to walk. As noted
in the Nature article, dogs in such cases usually die
around one year of age as a result of the failure of respiratory
muscles. In their experiment, researchers found that stem cells
successfully established themselves in the host tissue allowing
for the production of dystrophin, off-setting the effects of
muscular dystrophy. The injected stem cells not only produced
dystrophin in the effected leg, but in other areas of the body
as well, including respiratory muscles leading researchers to
believe that they are on the right track to finding an effective
treatment for MD sufferers.