Cell Therapy Slows Progression Of An Inherited Neurological Disease; Improves Mo

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Cell Therapy Slows Progression Of An Inherited Neurological Disease;

Improves Motor Skills In Mice

http://www.medicalnewstoday.com/medicalnews.php?newsid=40143

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 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 s 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.