Stem Cell trial for rare and fatal disease

The phase I trial for stem cells being used with Batten's disease is now taking place in humans. Batten's is a very rare neurodegenerative disease that affects children from 6 months to 10 years of age. These children lack an enzyme that breaks down complex fat and protein complexes. When these fats and proteins aren't broken down, they accumulate and eventually kill the cells around them becoming fatal by the late teens or early 20's. A phase I trial is not used to test effectiveness of the treatment, but rather the safety of the treatment, periodically testing on six different children with Batten's. In animal testing, the stem cells formed into cells that produce the missing enzymes. Researchers hope that this same outcome will occur in human testing. This is one of few tests proceeding to human testing after President Bush limited federal funding for stem cell research in 2001. As of that date, only embryos taken before the limit was placed could be tested on, which greatly reduced the amount of high quality testing. Hopefully this will be a step in the right direction for the children that are affected by this deadly disease.

http://health.msn.com/healthnews/articlepage.aspx?cp-documentid=100145773