OSAKA -- Japanese researchers report taking their first steps toward possible stem-cell-based treatments for muscular dystrophy.
A Kyoto University team and chemical company Asahi Kasei created skeletal muscle stem cells from induced pluripotent stem cells, or iPS cells. These were injected into a leg of a mouse with muscular dystrophy. In the muscle there, researchers detected a protein that kept muscle mass from deteriorating.
Japan has nearly 30,000 patients with muscular dystrophy.
The team, led by Kyoto University lecturer Hidetoshi Sakurai, says the stem cells fused with impaired muscles and helped regenerate them. This could lead to a therapy to slow the course of the disease. The researchers hope to ensure safety and effectiveness through testing on dogs and are eventually aiming for clinical trials.
At the National Center of Neurology and Psychiatry, a research team headed by Shinichi Takeda is also working on muscular dystrophy. The group has developed a cell transplantation technique. Muscle stem cells were created from iPS cells and shaped into globules.
Globular cells measuring 500 microns were then transplanted into a mouse with muscular dystrophy. Some impaired muscle was restored, according to the researchers. And three weeks later, the muscles were found moving inside the mouse. Takeda's team will also test the technique on dogs as early as next year before launching clinical trials.
Both teams will make presentations at a Japanese Society for Regenerative Medicine conference opening Thursday in Yokohama.