Giving Stem Cells a Chance

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Science  10 Jun 2005:
Vol. 308, Issue 5728, pp. 1519
DOI: 10.1126/science.308.5728.1519d

Many important issues surround stem cell and gene therapies, including at what time after birth treatments should be assessed and implemented. This timing is influenced by how early a disease manifests and whether it is considered sufficiently severe to warrant the risk of early intervention.

Escolar et al. provide an example of how very early stem cell therapy can enhance the chance of success. The study group consisted of newborns suffering from Krabbe's disease, a rare genetic disorder in which loss of a lysosomal enzyme in cells resident in the central nervous system allows the lipid substrates to accumulate, which results in severe neurological deterioration and death. In an attempt to correct this deficiency, stem cells from banked umbilical cord blood of unrelated donors were transplanted into newborns who either had already started to develop symptoms (142 to 352 days old) or had a family history of the disease but were as yet asymptomatic (12 to 44 days old). In the latter group, survival and neurologic development were significantly improved for almost all graft recipients, with cognitive functions in the normal range. Presymptomatic therapy in this case is likely to have allowed more efficient stem cell replacement of defective resident cells, thus avoiding some of the early toxic effects of the lipid substrates on young neurons and immature neural tracts. — SJS

N. Engl. J. Med. 352, 20 (2005).

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