Stem cell tests offer muscular dystrophy hope
LONDON (Reuters) - A new stem cell therapy that has enabled dogs with muscular dystrophy to walk more easily could be developed into a treatment to help humans with the disabling illness, Italian scientists said on Wednesday.
After transfusing a specific type of stem cell into the bloodstream of dogs with the genetic disease that causes progressive paralysis, they found improvements in the animals.
"We hope this could be a successful therapy (in humans)," said Professor Giulio Cossu, of the San Raffaele Scientific Institute in Milan.
"Even though by itself it may not lead to a complete cure, it would ameliorate the condition and then step by step we could work on this to the point of getting a real cure," he told Reuters.
After more tests on dogs, Cossu and his team hope to begin trials of the stem cell treatment in humans in a few years’ time.
Duchenne muscular dystrophy, which primarily affects boys, is caused by the absence of a protein called dystrophin that is involved in maintaining muscles. It is a progressive illness that usually begins between 3-5 years old. By the age of 12 many sufferers are unable to walk.
Drugs are given to slow the deterioration of the muscles and to control seizures but there is no therapy to stop or reverse the illness.
MAJOR STEP FORWARD
Cossu and his team tested the treatment on dogs with a disease similar to Duchenne muscular dystrophy following earlier studies on transgenic mice.
They extracted cells called mesoangioblasts, which are programmed to develop into muscle cells, from small blood vessels in the ill dogs. After inserting a normal version of the mutated gene, they replaced the cells in vessels in the dogs’ hind legs.
They also transfused stem cells taken from healthy dogs into ill dogs and gave them immune-suppressing drugs so they would not reject the donor cells. After five monthly injections, muscle function was restored in varying degrees and dystrophin was produced in cells in the animals. Dogs that received the donor cells showed more improvements in movement.
"Cossu and his colleagues’ results provide compelling evidence that this method should be developed further for testing in patients," said Jeffery Chamberlain, of the University of Washington in Seattle, in a commentary in the journal.
He added that there are more than 20 types of muscular dystrophy, as well as other muscle disorders, but few treatment options.
"Perhaps these conditions will be among the first for which the promise of stem-cell technology for degenerative disorders can be realized," he said.
Professor Dominic Wells, of Imperial College London, described the findings published online by the journal Nature as a major step forward in demonstrating the potential of stem cell therapy to treat the condition.
"This is the first piece of research that has convinced me that stem cell therapy could play a role in treatment for Duchenne muscular dystrophy," he said.
