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| Stem Cell Trial to Combat Childhood Brain Disease |
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| Batten Disease | |||
| Dienstag, 26. September 2006 um 00:47 Uhr | |||
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There are no translations available. Scouce: NewScientist.com By Helen Phillips The first clinical safety trial of a purified human fetal stem cell product is about to begin in the US for a rare and fatal childhood brain disease. The trial could pave the way for neural stem cell transplants to treat a range of brain and spinal cord disorders. A team from the Oregon Health and Science University Doernbecher Children's Hospital plan to treat six children suffering from the inherited neurodegenerative condition, Batten's disease - also known as neuronal ceroid lipofuscinosis (NCL). There is currently no alternative treatment for the disease. The team expect to treat the first child before the end of 2006. The children will receive injections of neural stem cells that have been purified - isolated from other cell types - and grown from donated human fetal tissue. The stem cell product and isolation technique was developed by StemCells Inc, of Palo Alto, California, which is sponsoring the trial. Children with Batten's disease suffer seizures, motor control disturbances, blindness and communication problems. As many as 600 children in the US are currently diagnosed with the condition - death can occur in children as young as 8 years old. The children lack an enzyme for breaking down complex fat and protein compounds in the brain, explains Robert Steiner, vice chair of paediatric research at the hospital. The material accumulates and interferes with tissue function, ultimately causing brain cells to die. Neuron support Previous tests on animals demonstrated that stem cells injected into the brain secreted the missing enzyme. And the stem cells were found to survive well in the rodent brain. Once injected, the purified neural cells may develop into neurons or other nervous system tissue, including oligodendrocytes, or glial cells, which support the neurons, say the researchers. Steiner is hopeful that the treatment will work for the 25 or so other hereditary brain diseases related to Batten's disease. In addition to secreting enzymes, Steiner says these cells can become the type of nerve cells found in spinal cord, and so they could potentially help after spinal cord injury. The stem cells can form into neural cells found in the brain or nerve cells found elsewhere in the central nervous system, he explains. However, Stephen Minger, director of the stem cell biology laboratory at Kings' College London, believes that despite Steiner's claims about the versatility of the new purified cells, their use is limited to Batten's disease. "The cells in question have little clinical relevance to other neurological disorders," he says. "Groundbreaking process" The researchers hope that the treatment will offer some clinical benefit to the children suffering from Batten's disease, but they stress that the primary purpose of the trial is to assess the safety of the product. It is the first such safety trial to be approved by the FDA. "This is a very important first step - a groundbreaking process to bring this technology to patients," says Nathan Selden, head of the Division of Paediatric Neurological Surgery at the hospital, who will perform the transplants. Human fetal stem cell transplants have been performed before on adult patients with neurodegenerative conditions including Parkinson's and Huntington's diseases, and also for spinal cord injury. But these used mixtures of various, unpurified fetal stem cells. Results have been mixed. The researchers plan to follow the children's progress over the course of a year.
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| Zuletzt aktualisiert am Sonntag, 21. Dezember 2008 um 11:09 Uhr |