a demonstration that stem cell efficacy could be enhanced even without the need for genetic engineering. (The drug, a glycosphingolipid biosynthesis inhibitor, is in a class of compounds called "substrate reduction therapy" drugs.) This part of the study not only represented the first "multidisciplinary" use of stem cells against a degenerative disease, but also highlighted the fact that, in the future, the most successful therapies - including those employing stem cells -- will likely invoke the use of multiple strategies in concert. Indeed, the stem cell may be the "glue" that ultimately holds these therapies together in an effective manner by virtue of its fundamental biology.
The researchers then sought to extend their insights to the use of human stem cells - either stem cells turned into neural progenitors from human embryonic stem cells - or isolated directly from the nervous system (called "adult" stem cells to distinguish them from embryonic stem cells even though they are taken from developing brain tissue). Both types of human stem cells were actually somewhat more effective than the mouse neural stem cells. And, they were equally as good as each other - in the first head-to-head comparison ever done between embryonic and "adult" stem cells, although the embryonic stem cells were somewhat easier to "scale up" into large quantities. Both types of human stem cells invoked the same range of multiple, collaborative mechanisms.
Neither type of human stem cell created tumors, deformation, a worsening of symptoms, or gave rise to inappropriate cells types. Neither cell type was rejected by the immune system. In fact, no immunosuppression was needed at all. Finally, the human embryonic stem cells were grown without mouse feeder layers and in a "defined" culture medium that is compatible with clinical use and demonstrating for the first time that such preparations are consistent with a therapeutic impact.
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