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A hope to dissolve autism's dark dreams
Parents' trust in secretin is put to test
Nov. 1, 2000
By Anita Manning, USA TODAY
Researchers are taking another look at a therapy
for autism that some believe is a miracle and others
say is a mirage.
In a clinical trial at five medical centers, doctors are trying to find out
whether the hormone secretin can mitigate symptoms of autism, a
developmental disorder that strikes an estimated one in 500 children by age
3, robbing them of the ability to interact normally with other people.
Some parents, convinced that secretin is all that stands between their
children and the relentless progression of the disorder, say they cannot wait
for the results. They're taking matters into their own hands, bringing secretin
into the USA and using it in ways that are not approved by the Food and
Drug Administration.
Autism often causes children to lose the
ability to speak or make eye contact,
and it is marked by bizarre repetitive
behaviors. Parents say their children
seem to fade before their eyes,
withdrawing into a world of their own.
"She had speech at 11 months — she
was saying 'Barney,' 'Mama,'" Sherry
Deese Jager says of daughter Megan,
now 4. Shortly after Megan's first
birthday, the girl began to change. Her
vocabulary of about 15 words declined,
and she started having gastrointestinal problems, a physical symptom
common to at least 25% of autistic children.
"After two years, her behavior started to snowball. At 2 years, 7 months,
she had stopped saying 'Mama.' She started jargoning (emitting guttural
sounds), making animal noises, all this ugly autistic stuff."
There are no drugs specifically designed to treat autism, a lifelong disorder,
though there are medications to ease some symptoms, such as
obsessive-compulsive disorder. The standard treatment for autistic children
and adults involves intensive speech therapy and individualized education.
The Jagers, who live near Annapolis, Md., had heard that some children
with autism improved — dramatically, according to some reports — after
being injected with secretin, a hormone that stimulates the pancreas to aid
digestion. Even though several small studies have found that secretin is no
better than a placebo in improving autism symptoms, the parents, desperate
for something to help their daughter, took her to a doctor who injected her
with the hormone. It was April 1999, and Megan was 3 years, 3 months
old.
"Four hours later, she spoke the words 'Let go,'" her mother says. "It was
amazing. She'd never said two words together."
It didn't stop there. Megan seemed to perk up and even began to interact
with others in ways she hadn't.
The little girl was given three more infusions of secretin in the next four
months. "After we did the infusions, Megan's behaviors improved," Jager
says. "It was effortless to do things with her. She was more compliant, more
tolerant. She slowly started losing all her obsessive-compulsive disorders."
Megan began attending a regular preschool part time during the summer and
will enroll in a regular kindergarten next year. "I never thought it would be
possible," her mother says.
Walter Herlihy, president and CEO of Repligen, a Needham, Mass.,
biotech company that makes synthetic secretin, believes that the hormone
acts on the brain by stimulating a nerve that connects the digestive tract to
the brain. The nerve signals the pancreas to release juices that neutralize
stomach acid, he says. Secretin "acts through a known brain pathway," he
says. "It may well be that in stimulating that part of the brain, other
neurotransmitters are being activated."
However it may work, Jager says, the effect on Megan has been "very
miraculous."
"Her vocabulary is improving, and the (autistic) behaviors have decreased.
This treatment has allowed my daughter's autistic condition to go into
remission," she says. "She has awakened."
Jager is quick to say that secretin is not a cure and doesn't work for all
children. But she believes without question that it helps some children and
that Megan is one of them.
"I would like every autistic child to have the opportunity to try secretin," she
says.
That can't happen any time soon. Secretin is approved by the FDA only to
diagnose pancreatic problems. Ferring Pharmaceuticals of Tarrytown, N.Y.,
manufactured it in a form taken from pigs, but Ferring ceased production in
April 1999, citing low demand for it as a diagnostic agent.
Since then, a synthetic secretin has been available in the USA only through
clinical trials. Jager won't say where she gets her supply, but doctors say
many parents stockpiled it when the manufacturer stopped production, and
others are bringing it in from overseas.
"They're importing secretin from Japan or buying it off the Internet," says
researcher Cindy Schneider of the Southwest Autism Research Center in
Phoenix, one of the five sites involved in the clinical trial.
Some parents mix it with a solvent and apply it to their children's skin in the
belief that it can be absorbed.
"Chemists will tell you it's a very unstable compound, and they doubt it
would be absorbed intact," Schneider says. "Some give it in sublingual
(under the tongue) drops, and some families swear that helps their child. I
can't tell you if it's (usable by the body) that way or not. You have a lot of
desperate parents. This is a medical experiment on a grand scale."
Repligen holds the patent on secretin for use in autism therapy and is
sponsoring the current clinical trial, which Herlihy says is the largest study to
investigate the hormone in autism. Results should be available early next
year. About 140 children, ages 3 to 6, are being given either secretin or a
placebo — even the researchers won't know who got which until the study
is completed — to determine whether secretin helps any of them, and if so,
which ones.
Marie Bristol-Power of the National Institute of Child Health & Human
Development says the Repligen study, in combination with earlier trials, will
provide "very good data to support this or put it to rest."
So far, she says, "the data have not been promising," but "the research has
been successful in stimulating more interest in the gut-brain connection and
finding out if secretin isn't a cure, is it a clue to what's going on in at least a
subset of children?"
Max Wiznitzer, pediatric neurologist and head of the Autism Center at
University Hospitals in Cleveland, believes that secretin has no effect on
autism, but it may make children feel better as their gastrointestinal problems
are addressed.
Possibly, he says, "the secretin fixes whatever the problem is with the gut,
and as the gastrointestinal system feels better, so do they."
"It's not a fix for autism," he says. "The core issue is what's going on with the
gut." He suspects that autism has something to do with a brain chemical
called serotonin, which also is found in the digestive tract, and says that
more research is needed.
Meanwhile, Wiznitzer says, autistic children who have gastrointestinal
problems should be evaluated and treated.
Those cases in which behavior seems to improve may be coincidental, he
says. "How do you know the kid wasn't going to start talking anyway?
"The average age when (autistic children) start talking is 3, so that's going to
happen anyway. You can't sort it out from the background noise. There's
much more that has to be done to prove a causal relation."
Schneider says the Repligen study is designed to be more specific than
earlier studies.
"We have a larger number of children, but also we're using measures that
are less open to interpretation," she says.
In the study — being carried out at the Mayo Clinic; the Rochester Institute
for Digestive Diseases; the University of Maryland Medical Center; the
University of California, Davis/MIND Institute; and the Southwest Autism
Research Center — only severely autistic children with gastrointestinal
symptoms are enrolled, Schneider says.
They're being given three doses of secretin over 10 weeks. "We're going to
learn a lot about a specific subgroup of children with autism," Schneider
says. "We're not comparing apples and oranges. We're just looking at the
oranges. That has to be the future of autism research."
Herlihy says that if secretin helps even a tiny fraction of children with autism,
that's worth celebrating. "If we're going to wait for a drug or treatment to
work on everybody," he says, "we're going to wait a long time."
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