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Stem cell trial for fatal childhood brain disorder is set to start

2 October 2006
Appeared in BioNews 378

The first trial for a proposed stem cell treatment for Batten's disease is about to begin. Researchers at the Oregon Health and Science University's (OHSU) Doernbecher Children's Hospital, US, plan to treat six children with the rare neurodegenerative disorder by using fetal stem cell transplants.

Children with Batten's disease cannot produce the normal amount of a particular 'lysosomal' enzyme. This enzyme is needed to break down the fat and protein particles in the brain, known as lipopigments, which are toxic when they build up in the body's tissues. 'These materials accumulate and interfere with normal cell and tissue function and ultimately cause cells to die', explains Dr. Robert Steiner, the lead researcher and vice chair of paediatric research at Doernbecher Children's Hospital.

The team plans to take neural stem cells developed from fetal tissue by StemCells Inc of Palo Alto, California, and implant them into the children's brains. The researchers hope that these transplanted cells will develop into specialised cells that will produce the correct amount of the lysosomal enzyme needed by those with Batten's disease. If the clinical safety trial is successful, it may mean that stem cell transplants could one day eliminate the debilitating conditions associated with Batten's, such as blindness, communication problems, worsening seizures, and the progressive loss of body movement.

Batten's disease is a juvenile form of Neuronal Ceroid Lipofuscinosis (NCL), which first appears between the ages of 5-10. There are three other types of NCL: two that begin at four years old or younger, and a very rare form that affects adults. According to the National Institute of Neurological Disorders and Stroke (NINDS), Batten's disease is rare and occurs in just two to four of every 100,000 live births in the US, but it is more commonly found in families that have an inherited gene for the disorder. Death from Batten's disease can occur as young as eight years old, although the majority of patients survive until their late teens or early twenties.

The scientists expect to have treated the first child before the end of 2006 and, if everything goes smoothly, will then start treating the remaining five. 'We will begin with one patient, but we are not going to continue on with other patients until all of the safety protocols evaluated in the first patient have been met', says Dr. Nathan Selden, head of the Division of Pediatric Neurological Surgery at Doernbecher and OHSU's School of Medicine.

The team also plan on following up all six children for a year after they are treated to check for any long-term side effects. Steiner tells us that although he hopes that this trial will provide an insight into a potential treatment option for Batten's disease, he cautiously points out that it is early days yet. 'This trial is just the beginning in a lengthy, ongoing effort to determine a safe and effective means to improve the quality of life of those suffering from NCL', he says.

Stem-Cell Trial for Neurodegenerative Pediatric Disease to Get Under Way |  22 September 2006
Stem cell trial to combat childhood brain disease
New Scientist |  25 September 2006
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