Low levels of nerve growth factor in cerebrospinal fluid of children with Rett syndrome.

A role of neurotrophic factors has been postulated in some human neurodegenerative disorders such as Alzheimer's and Parkinson's disease. The known developmental effects of these substances suggested that, in some neurologic diseases affecting children, neurotrophic factors might be inadequate. Using a sensitive, two-site enzyme-linked immunoassay, we examined the content of nerve growth factor in the cerebrospinal fluid of 11 children with Rett syndrome and of 24 control patients with various neurologic diagnoses or suffering from other diseases. Nerve growth factor levels were significantly lower in the cerebrospinal fluid of the patients with Rett syndrome than in control patients. The lower level of cerebrospinal fluid nerve growth factor in Rett syndrome suggests that lack of nerve growth may be involved in the pathogenesis of this disease or reflect the underlying brain damage present.