Fact Sheets 278

Autism is a developmental disorder first seen in childhood and is characterized by difficulty with social interaction and communication and by unusual forms of repetitive behavior. Most clinical interventions address the symptoms of the disorder, with conflicting reports on their usefulness.

Because of a lack of success with treatments for the brain damage itself, unproven treatments for the basic cause(s) are often tried but to date have not proven to be effective.

Recently a story was disseminated by the news media about a three-year-old child with autism who had a dramatic improvement within a week after a single dose of the intravenous administration of a hormone: secretin. This has been followed by other news reports of similar improvement in other children with autism. Secretin is a hormone important for digestion; it is released by the gut (duodenum) and stimulates the pancreas. It has been approved by the FDA for use in the diagnosis of gastrointestinal disorders. Secretin is available for this diagnostic purpose as a purified compound derived from the gut of pigs or as a human variety synthesized in the laboratory.

In order to test the usefulness of human synthetic secretin in the treatment of autism in children, a carefully designed (double blind; placebo controlled) clinical trial was conducted and the results have been reported.¹ The trial evaluated the effect of a single dose of secretin in 60 children (ages 3-15) with autism or pervasive developmental disorder (PVD). PVD is a diagnosis used when the child does not meet all the criteria of autism, but has some of the major symptoms. The children were equally assigned by the diagnosis (40 autism; 20 PVD) and to treatment by either secretin or a placebo (an inert compound). The children were evaluated prior to treatment, on the 1st and 2nd days after treatment and one, two and four weeks after treatment.

Evaluations were done by trained clinical personnel, by the childs parents, and by the childs school or preschool teacher.

The study demonstrated that "a single dose of synthetic human secretin is not an effective treatment for autism or pervasive developmental disorder".

Comment:

Developmental disorders, particularly of the nervous system (e.g. mental retardation; cerebral palsy; autism) serve as a magnet for the use of unsubstantiated "treatments". Although individual experiences with a "new treatment" very properly serve as a basis for stimulating a carefully designed clinical evaluation, individual experiences do not in themselves serve as proof of the usefulness of the "new intervention." We are grateful that these investigators moved rapidly to scientifically evaluate the usefulness of secretin as a treatment for autism. It is fortunate that we now know that a single dose of synthetic human secretin does not have positive results. Would several doses give better results? Would naturally occurring animal or human secretin give a better result than the synthetic human variety? Should the evaluation be done for longer periods of time (a long term effect)? All of these are reasonable questions; however, the question about the immediacy of the effect has been answered: Synthetic human secretin doesn't work except for a short-term placebo type effect in some children.

¹ Sandler AD et al. Lack of Benefit of Human Secretin in the Treatment of Autism and Pervasive Developmental Disorder. NEJM 1999; 341": 1801-1806