Stem Cell Treatment for Batten Disease

Batten Disease is an uncommon, fatal neurodegenerative disorder that begins in childhood. 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 be 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 suffer mental impairment, 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. Batten disease is a life limiting disease, Life expectancy for sufferers of batten’s disease varies. According to the National Institute of Neurological Disorders and Stroke, there is currently no treatment for the disease. It usually becomes fatal by the late teens or early 20s.

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.