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 I