Dec. 5, 2008

Neural Stem Cells Help SMA Mice

Mice with a disease resembling human spinal muscular atrophy (SMA) that were treated with specialized stem cells lived 39 percent longer and showed better neuromuscular function than did untreated mice, lending support for development of this type of therapy for SMA.

Investigators coordinated by Giacomo Comi at the University of Milan (Italy), who published their findings in the October issue of the Journal of Clinical Investigation, isolated neural stem cells, which can become motor neurons under certain conditions, from the spinal cords of mouse embryos not affected by SMA.

After exposing these cells to growth-promoting substances and other chemicals and allowing them to multiply in the laboratory, the researchers injected them into the spinal fluid of 1-day-old SMA mice.

The donor cells migrated into the spinal cords of the SMA mice, where they generated cells that appeared to be motor neurons (the muscle-controlling nerve cells that die in SMA) and may also have aided existing motor neurons in their struggle to survive.

"It is conceivable," the authors note, "that in the future, stem cell transplantation could be combined with other molecular and pharmacologic approaches to achieve an effective recovery."