Updated: Monday, 20 Apr 2009, 6:57 PM EDT
Published : Monday, 20 Apr 2009, 4:29 PM EDT
(ABC) - Researchers have discovered a potential drug for the most common form of Muscular Dystrophy.
They're using a protein already found in the human body to combat muscle damage.
It's been 20 years since scientists discovered the gene that causes the most common type of Muscular Dystrophy -- Duchenne MD. And patients are still waiting for a cure.
"The mutation causes muscle fibers to pull away from each other and with progressive use of your muscles in these patients it eventually leads to muscle damage, severe muscle damage," said Dean Burkin of the University of Nevada.
Gene therapy might be a solution, but researchers at the University of Nevada in Reno are excited about a simpler, and potentially safer, approach.
"This could be an IV drug for the patients, if the work in the mouse models that we've been using translates to human studies," Burkin said.
Researchers have discovered a protein called Laminin-111. It's something the body normally only produces before birth and helps keep muscle fibers from deteriorating.
They tested it in muscle cell samples from human patients and in mice with MD and found exercise caused muscle damage in the untreated mice, but not in the treated animals.
"And so this was a significant finding suggesting that Laminin-111 was really acting protective in the muscle to prevent it from degeneration," said Burkin.
The researchers also tracked the protein and were surprised to find it reached all major muscle groups in the mice.
"Including the heart and the diaphragm which are most severely affected in Duchenne patients," said Burkin.
The mice in the study were treated before they began to show symptoms of Muscular Dystrophy.
So the next step for scientists is to find out if the therapy can halt the disease's progression after it starts.
The University of Nevada has licensed its patent for the protein to a Boston company which is now working to make a human version for possible clinical trials.