CHAPEL HILL -- Parents will go to great lengths to help children with illnesses, including trying the latest well-publicized treatments regardless of whether they've been proven effective. But if a therapy sounds too good to be true, it usually is.
A new study appearing in the Dec. 9 issue of the New England Journal of Medicine offers a prime example. Contrary to what early publicity suggested, North Carolina researchers found no evidence that a single dose of the synthetic human hormone secretin benefited children with autism, an often severe developmental disorder.
In a well-controlled study of 60 children, half of whom received synthetic secretin and half of whom received an inactive salt solution, scientists could tell no difference between the groups when the study ended. The investigation was double-blind, meaning that neither clinicians nor parents knew which of the two treatments the children received until evaluations were complete.
"Following news reports, many parents contacted us wanting to have their children treated with secretin," said developmental pediatrician Dr. Adrian Sandler. "We felt the responsible thing to do was to enroll these children in a carefully controlled and well-designed study first.
"Some individuals in both groups showed a variety of positive responses in terms of symptoms and associated behaviors," he said. "But because there were no overall group differences, the study points to a placebo effect. Families of children with autism were excited and expected some kind of treatment benefit, but we saw no benefit."
Sandler is medical director of the Olson Huff Center for Child Development at Thoms Rehabilitation Hospital in Asheville, N.C., and clinical associate professor of pediatrics at the University of North Carolina at Chapel Hill School of Medicine. He conducted the study with Dr. James W. Bodfish, associate director of the Human Development Research and Training Institute in Morganton, N.C., and clinical associate professor of psychiatry at UNC-CH, and other colleagues.
Bodfish said intense interest in secretin arose early this year following a "Dateline NBC" report describing almost miraculous results in an autistic child from a single treatment with the hormone.
Several newspaper stories, including one in The Wall Street Journal, followed and boosted interest.
"It's disappointing that we didn't find any treatment effect, but the good news is that we were able to mobilize the clinical and research communities to answer fairly quickly in a large group of children the burning question, 'Does this medication work?'" he said. "Next we have to use this momentum to continue the search for effective and safe treatments for autism.
Bodfish said that other groups also funded by the National Institute of Health are evaluating other forms of secretin treatment.
"Early indications are that their results are the same as ours," he said.
Children in the N.C. study ranged in age from 3 to 14 and were randomly assigned to either secretin or placebo groups, Bodfish said. Following treatment, they were evaluated with standardized measures involving parents and doctors rating children's behavior and symptoms.
Autism is a rare but frequently devastating condition that usually becomes apparent within the first year of life, the researchers said. Three or more times more boys are affected than girls, and both the cause and effective treatments approaching cures have eluded scientists.
Victims of the illness, which is believed to be partly genetic, tend to withdraw from contact with others, including parents. They also tend to behave oddly and develop language and other social skills far more slowly than normal. Severely affected patients can never lead independent lives and may need to be institutionalized. Strains on families often are enormous. With special attention and schooling, however, patients with milder cases can fare reasonably well.
Medical scientists need to scrutinize all potentially better therapies carefully, Sandler said.
"We have a responsibility to maintain a commitment to evidence-based medical practice and to be open-minded in looking at a variety of treatment options," he said. "There already are some proven treatments for children with autism that include behavioral and educational approaches pioneered at the University of North Carolina at Chapel Hill. I think that in the next 10 years we are going to see real breakthroughs in our understanding of autism and possibly in treatments as well."
Others involved in the new study were pharmacist Jeffrey DeWeese, nurse practitioner Mary Alice Girardi and pediatrician Dr. Victoria Sheppard, all at Thoms Hospital, and research assistant Kelly A. Sutton of the Western Carolina Center, an affiliate of the N.C. Mental Retardation Research Center at UNC-CH.
Note: Bodfish and Sandler can be reached at 828-438-6518 and 828-274-6189, respectively.
Contact: David Williamson, 919-962-8596.
Journal
New England Journal of Medicine