In Clinical Trial, Hormone Shows No Effect on Autism

A hormone that has been highly touted as the first effective drug treatment for autism showed no benefits for children with the mysterious disorder in the first of several government trials, North Carolina researchers report today.

The drug, secretin, is a hormone that the body naturally produces to assist in digestion. It has been in great demand since a New Hampshire woman reported last year that her 3-year-old autistic son dramatically improved after receiving secretin during a diagnostic procedure for stomach disorders.

That report prompted many parents of autistic children to travel overseas or pay high fees--in some cases even mortgaging their houses to do so--to get the scarce drug for their children.

But the North Carolina researchers report in today’s New England Journal of Medicine that 28 children receiving secretin injections showed no more improvement than 28 others who received a placebo. Another study by the University of Chicago, posted on the Internet, reports similar results.

“These findings strongly suggest that secretin should not be recommended to treat autism until the results of other ongoing studies are known,” said Dr. Duane Alexander, director of the National Institute of Child Health and Human Development.

Reaction among parents of autistic children was mixed. Dr. Bernard Rimland of the Autism Research Institute in San Diego downplayed the finding, adding that “there are at least 13 or 14 other studies of secretin in the pipeline that I know of. This is just the first half of the first inning.”

But Portia Iversen, president of the Los Angeles-based organization Cure Autism Now, suggested that the initial reports on secretin were overblown. The original researchers, she said, “should have dissected the original report [about secretin] more carefully” to determine if the hormone was, in fact, the responsible agent. “It did surprise me that so much research took place based on one incident.”

Autism is a severe developmental disorder in which children seem isolated from the world around them. It is marked by poor language skills and an inability to handle social relations. No cure exists, but many problems can be alleviated with intensive behavioral therapy.

The conventional wisdom is that about one in every 2,000 children suffers from autism. Several recent studies, however, suggest that the proportion is growing and may be as high as one in every 500 in many communities. In some places, such as Brick Township, N.J., for example, parents say the incidence is closer to four cases for every 500 children.

The cause of autism is unknown, although psychologists are emphatic in stating that it does not reflect poor parenting skills, as was once believed. It seems clear that genetics plays a role in susceptibility, at the very least, and researchers have identified small segments of at least two chromosomes where they suspect that the genes involved may be hiding.

A group of parents who believe that autism is an autoimmune disorder triggered by childhood vaccinations has undertaken a vociferous campaign against such injections. But there is no proof that validates their theory.

One Boy’s Experience Ignites Interest

The secretin issue evolved from the susceptibility of many autistic children to diarrhea and other gastrointestinal disorders.

That link burst into public consciousness last year when Victoria Beck took her son Parker to see Dr. Karoly Horvath of the University of Maryland Medical Center because of the boy’s autism-linked gastrointestinal distress.

Horvath administered secretin to Parker as part of his standard diagnostic procedures. In the following days, Beck noticed a dramatic reduction in her son’s symptoms. Parker’s digestive problems improved, he began sleeping through the night and he even began saying words. She persuaded Horvath to follow up on her observation, and the doctor subsequently published a report on three children who seemed to improve after being treated with the drug.

The University of Maryland filed for a patent on the use of secretin for treating autism but, after a bitter dispute, signed the patent over to Beck because she was the first to suspect a connection. It is very unusual for a non-scientist to hold such a patent.

After the television show “Dateline” aired a report about her son, parents all over the country began asking doctors to prescribe the hormone for their children.

“Many parents have a sense that their child is locked up in there,” Iversen said, “and that drives them to try to save the child. . . . Anything that comes along, we feel that we have to explore.”

Secretin is extracted from the duodenum (part of the small intestine) of pigs, and the only approved source was a Scandinavian laboratory that stopped producing it last spring. The biotech company Repligen began making it through recombinant DNA procedures. The firm has not yet received approval to market the recombinant version of the drug.

Consequently, there have been reports of some parents mortgaging their houses to purchase the drug or of hoarding supplies in their freezers. The drug now costs about $185 for a small vial.

Alarmed by the widespread, unapproved use of secretin, the National Institute of Child Health and Human Development quickly organized clinical trials of the drug. The new results are the first reported from that program.

Psychologist James W. Bodfish of the University of North Carolina and his colleagues found some improvement among the autistic children getting the drug, but an equal improvement among those getting the placebo.

“We have been unable to show any benefit from the secretin treatment,” said his colleague, Dr. Adrian D. Sandler of Thoms Rehabilitation Hospital in Asheville. The strong placebo effect probably arose as a result of the extra attention the children received from both their parents and the physicians, he said. Such effects are common in autism trials, Iversen added.

Bodfish pointed out that the child health institute has several other trials underway as well, but said, “Early indications are that their results are the same as ours.”

Rimland of the Autism Research Institute said the results of the study are somewhat suspect because the researchers used psychological examinations that are designed for diagnosing autism but are not really appropriate for assessing the effects of therapy. And, he added, 70% of the parents asked that their children continue to receive secretin even after its failure in the test was explained to them.

Rimland remains optimistic because of the strong anecdotal evidence of secretin’s effects.

“Immediately after getting secretin, kids who never had a normal bowel movement have one,” he said. “Kids who never slept the night through sleep the night through. Kids who have never made eye contact with their parents do so.

“These sudden, dramatic changes are not just figments of their parents’ imaginations. They were just not picked up in these studies, for some reason.”