Stem cell treatment successful in mice with brain disorder

Scientists from the Burnham Institute for Medical Research in California, US, have shown that stem cells taken from embryos and fetuses can delay the progression of a fatal brain and nerve disease in mice, by repairing damaged neurons in the brain. The study, published in Nature Medicine, demonstrated that treated mice lived seventy per cent longer than the control group, and although the disease eventually returned, the scientists say providing booster injections of stem cells could prevent this from occurring. The use of mouse neural stem cells was compared with human neural ('adult') and embryonic stem cellc, with the human stem cells proving to be more effective. The study found no difference between the human adult and embryonic cells, although the embryonic cell treatment was easier to 'scale up'.

Dr Snyder, who led the international collaboration and is director of Stem Cells and Regeneration at Burnham commented, 'In fact, our study offers the first evidence that stem cells employ multiple mechanisms - not just cell replacement - which collaborate to benefit disease. These findings also raise the possibility - somewhat counter-intuitively - that stem cells may inherently exert an anti-inflammatory influence in degenerative diseases'.

The mice used in the study were bred with the equivalent of Sandhoff disease, one of several 'lysosomal storage diseases', which also include Tay Sachs disease. Infants affected by Sandhoff disease rarely live beyond the age of six. Fia Richmond, founder of Children's Neurobiological Solutions Foundation, welcomed the news. 'Dr. Snyder's team has extended the promise of stem cell therapies to children with special-needs, including those with Sandhoff disease', she said.

The researchers used cell lines approved for funding by the US Government, which under legislation enacted by President Bush, must have been derived before 9 August 2001 to be eligible for federal funding. Snyder plans to request permission from the US Food and Drug Administration to start human clinical trials in children, but proposes to use only fetal stem cells, and not embryonic cells. 'I think they are a little bit squeamish,' he said.