Stem Cell Treatment for Batten Disease

In November 2006, doctors at Doernbecher Children's Hospital at Oregon Health & Science University began a clinical study in which six children received stem cell treatment for Batten’s disease. The study participants were followed for six month. While this stem cell therapy has not yet provided a cure, the stem cell treatment itself appears to be safe.

Batten Disease, also called Neuronal Ceroid Liposfuscinosis, is an uncommon, fatal neurodegenerative disorder that begins in childhood. It is caused by a deficiency of a lysosomal enzyme, and it is a rare disease that affects an estimated 2 to 4 of every 100,000 babies born in the United States. It is a hereditary disease. The first symptoms usually appear around the ages of 4 to 10, starting with the gradual onset of vision problems or seizures. Other early signs may include slight changes in personality and behavior, slow learning or regression, repetitive speech, and clumsiness. There may be slowing head growth, poor circulation in legs and feet, curvature of the spine, a decrease in body fat and muscle mass, hyperventilation and/or breath-holding spells, teeth grinding, as well as constipation.
Eventually, these children become mentally impaired, the seizures worsen, and the child’s sight grows progressively worse, as do speech and motor skills. With time, children with batten disease become blind, bedridden, and demented. Life expectancy for sufferers of Batten’s disease varies.
Six genes have already been identified that cause different types of Batten disease in children or adults. Others are yet to be identified. It is known that two of these genes encode enzymes, however, the function of most of these genes remains unknown. Identifying the genes opens the possibility of gene replacement therapy or other gene-related treatments.
In October 2005, the FDA approved the transplantation of fetal neuronal cells into the brains of children suffering from infantile and late infantile versions of Batten disease. Then in November 2006, doctors at Doernbecher Children's Hospital at Oregon Health & Science University began a clinical study in which purified neural stem cells were injected into the brain of a six-year-old child with the disease. Prior to treatment, this child had lost the ability to walk and talk. The patient was the first of six to receive stem cell therapy. The study participants were followed for six month. While this stem cell therapy has not yet provided a cure, the stem cell treatment itself appears to be safe.
 

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