Cell therapy slows progression of metachromatic leukodystrophy

In an important discovery, scientists have demonstrated that the progression of a type of genetic brain disease is slowed and symptoms are improved in mice that received cell transplants.

The new study, published in the March 22 issue of The Journal of Neuroscience, may have implications for developing new therapies for metachromatic leukodystrophy, or MLD, a fatal, relatively rare inherited disorder that in humans usually begins early in life. 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 and from other areas of the body, resulting in symptoms including convulsions, seizures, personality changes, spasticity, progressive dementia, motor disturbances progressing to paralysis, and blindness. There is no cure; the only current treatment is a bone marrow transplant.

Ernesto Bongarzone, PhD, and his colleagues at the San Raffaele Scientific Institute in Milan, Italy, transplanted cells that produce myelin into the brains of newborn MLD mice. The researchers found that the transplanted myelin-producing cells survived in the mice brains 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.

"There is much excitement in the field of cell-based therapy and this study is a clear indication of its potential," says Mahendra Rao, MD, PhD, at Johns Hopkins School of Medicine and the Invitrogen Corporation. "This carefully conducted study suggests that a different cell type, such as myelin-producing cells, may be better than others when used for therapy."

"These results contribute to a growing field of intense research where the use of brain-derived cells, including myelin-producing cells and neural stem cells, may be envisioned as direct cell therapies to target specific neurological diseases," says Bongarzone.