Cell Therapy Slows Neurological Disease

Italian scientists have determined the progression of a type of genetic brain disease is slowed and symptoms improved in mice receiving cell transplants.

The finding by researchers at the San Raffaele Scientific Institute in Milan, Italy, might have implications for developing new therapies for metachromatic leukodystrophy, or MLD, a fatal, relatively rare inherited disorder.

In the disease, the fatty substance sulfatide accumulates in the brain due to the lack of an enzyme and causes loss of the white matter, or myelin, protecting nerve fibers. Without myelin, nerves cannot conduct impulses to other areas of the body, resulting in symptoms including convulsions, seizures, progressive dementia, motor disturbances progressing to paralysis, and blindness.

There is no cure; the only current treatment is a bone-marrow transplant.

In the study, Ernesto Bongarzone and colleagues transplanted cells that produce myelin into the brains of newborn MLD mice. The researchers found the transplanted myelin-producing cells survived and successfully moved to regions of the brain where they could aid in producing myelin. The transplanted cells also helped lead to production of healthy myelin and improved motor coordination.

The research appears in The Journal of Neuroscience.