Children with lethal inherited degenerative diseases of the
brain, the lysosomal storage diseases or LSDs, will be among the
first to benefit from novel approaches based on stem cell
therapy (SCT). This belief is based on a number of medical and
experimental observations including: 1) These diseases cause
profound mental retardation or lead to death in most affected
children; 2) SCT has already been shown to work in the milder
forms of LSD that do not affect the brain; 3) The clinical
safety of neurotherapy by stem cell transplantation is already
being established in the LSDs; 4) Many of the pertinent
regulatory hurdles have already been overcome; and 5)
Experimental work has clearly shown that SCT can be used to
treat the brain in the severe forms of LSD. This combination of
medical and experimental data strongly suggest that the LSDs are
uniquely poised to benefit from novel SCT and that this therapy
is likely to be successful, a prediction that can less reliably
be made for SCT applied to other brain and s