A company set to begin clinical trials of a stem-cell treatment for a fatal brain disease has announced that the treatment boosts survival in a mouse model. The company, StemCells of Palo Alto, California, received approval from the US Food and Drug Administration in October 2005 to use human neural stem cells to treat infants with neuronal ceroid lipofuscinosis, or Batten disease. Infants with this hereditary disorder lack enzymes to break down a chemical called lipofuscin. In those affected, the chemical kills brain cells, and eventually proves fatal.
StemCells plans to give newborns neural stem cells that make enzymes to break down lipofuscin. The neural stem cells, made from cells harvested from human fetuses, are more developed than embryonic stem cells, which can give rise to all the cell types of the body, but more flexible than adult brain cells. On 1 July, Nobuko Uchida, a scientist with StemCells, presented data on tests in mice at the meeting of International Society for Stem Cell Research in Toronto, Canada.
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