Health officials Wednesday urged parents of autistic children not to give them the hormone secretin after four separate studies found that the much-touted miracle cure was no better than saltwater at fighting symptoms of the brain disorder.
The largest of the double-blind studies, which measured the effects of secretin and a placebo in 56 children between the ages of 3 and 14, was published Wednesday in the New England Journal of Medicine. Secretin and saline solution both improved the behavior of the autistic children.
"These findings strongly suggest that secretin should not be recommended to treat autism until the results of our other ongoing studies are known," said Dr. Duane Alexander, director of the National Institute of Child Health and Human Development.
Autism is a devastating brain disease that usually has onset in infancy or early childhood, characterized by absent social skills and abnormal intelligence. It affects as many as 600,000 Americans and appears to be increasing, for reasons unknown.
In addition to their impenetrable personalities many autistic children have sleep problems and diarrhea. And gastrointestinal problems were what led the autistic community to secretin.
In April 1996, Victoria Beck brought her 4-year-old son Parker to a Maryland clinic to receive an infusion of secretin, a pig hormone used to test pancreatic function.
Within a few days of the diagnostic test, Parker underwent a miraculous transformation, his mother says. A mute boy with disrupted sleep and erratic bowels, he began talking and sleeping through the night. His diarrhea went away.
Beck, an enterprising New Hampshire housewife, had the perspicacity to videotape the changes in her son's behavior. She wrote a book about secretin, filed a patent for its use and appeared on "Good Morning America," "Dateline" and other TV shows. Soon thousands of parents were clamoring for the drug. According to Ed Purich, a pharmacist involved in secretin research, as many as 8,000 autistics have gotten infusions in the last two years.
The day after Beck's appearance on "Dateline," phones started ringing at pediatricians' offices around the country -- parents of autistics eager to try anything that would spark communication with their damaged children, who typically live in a foggy world of repetitive actions and strange obsessions, beyond the reach and understanding of their loved ones.
As it happens, the only company that produced pig secretin for the diagnostic market stopped making it, and supplies dried up. Unscrupulous salesmen have charged up to $15,000 per vial for products that are either illegally imported, derived from unfiltered industrial stocks or simply don't contain secretin at all.
Other parents, including Beck, have started administering secretin transdermally by mixing it with a substance called DMSO, which probably denatures the hormone before it can even be absorbed in the bloodstream. "It's really a waste of money," Purich says.
Beck and Bernard Rimland, a maverick autism advocate in San Diego, sold the secretin patent last year for a reported $1 million in cash and stock to Repligen, a Needham, Mass., biotech company whose CEO, Walter Herlihy, has two autistic children. (Beck donated her $750,000 in stock to Rimland's institute).
In January, meanwhile, the National Institutes of Health put out a request for double-blind studies of secretin. So far, groups in Atlanta, North Carolina and two in Chicago have presented data that has all shown no more benefit from secretin than placebo.
All the studies, however, showed some benefit from both placebo and secretin -- cementing the impression that secretin has no particular value other than as placebo.
"Some treatments are identified serendipitously and that was the hope with secretin," said James Bodfish of the University of North Carolina in Chapel Hill, whose study was published Wednesday in the Journal. "This is disappointing but the good news is that the clinical and research communities were able to quickly answer questions parents had."
Rimland and Herlihy deny that the Journal article is the last word in secretin. Rimland, who predicted earlier this year that 70 percent of autistics would benefit from secretin, said that study of the hormone is still in its early stages. The told-you-so attitude of many pediatric neurologists, he said, "is not surprising since this is a community that's negative about any innovation that doesn't include psychoactive drugs like Prozac and Ritalin."
Repligen's stock fell Tuesday amid word of the Journal article (the company also produces a purification device for making antibodies and has an immunosuppressant for leukemia patients in trials).
But Herlihy said he was confident a subset of autistics -- perhaps 20 percent -- would eventually benefit from secretin.
"That may not sound like much but this is a disease where nothing works. Twenty percent would make me the happiest man on earth." His children have gotten secretin, Herlihy says, and while "they haven't been reciting sonnets, they've been more social, they've reached up to the edge of the group."
He noted that all the trials reported so far gave only one dose of the hormone, which he said may work best with repeated doses.
Another physician with autistic kids, Phoenix obstetrician Cynthia Schneider, has also completed a controlled double-blind study of secretin. She, too found no statistical difference in the response of placebo and secretin recipients.
Schneider began studying secretin after her 8-year-old son, Derek, thrived on it. She says that individual patients within the study showed remarkable results from the hormone.
One 3-year-old who hadn't slept more than two hours in a row his entire life began sleeping through the night and taking an afternoon nap, she said. "His mother said, 'He went from being Tigger to Pooh.'"
She also said autistics with gastrointestinal problems did well in her study -- the theory being that these patients have a genetic abnormality, a shortage of natural secretin production, that is linked to both the brain and gastrointestinal problems.
Other researchers, including Bodfish's group, saw no particular benefit for the subgroup of diarrhetics in their study.
While a single dose of secretin seems to be safe -- only a small number of adverse reactions have been reported -- scientists are worried that repeated doses could cause autoimmune reactions, particularly when the pig hormone is used.