Alice Trinkl, News Director
Janet Basu (415) 476-2557
jbasu@pubaff.ucsf.edu
FOR IMMEDIATE RELEASE
May 13, 2000
LANGUAGE-TESTING STUDY SHOWS NO EFFECT
FROM SECRETIN FOR CHILDREN WITH AUTISM
Sometimes wishing does not make it so.
Parents of autistic children began calling Melvin Heyman, MD, chief of
pediatric gastroenterology at the University of California, San Francisco in
1998. They were full of hope about reports that secretin, a hormone produced in
the intestines and used in a diagnostic procedure, might improve their children'
s ability to talk and interact with others. To test that concept, Heyman and
clinical research fellow Jenifer Lightdale, MD, teamed with autism experts from
UCSF's Langley Porter Psychiatric Institute to perform objective tests of
language and behavior before and after administering secretin.
The results were reported May 13, 2000 at the Pediatric Academic
Societies/American Academy of Pediatrics meeting in Boston. Twenty autistic
children, aged 3-6, received formal language testing before infusion with
secretin and in four follow-up tests in ensuing weeks. No child showed
significant changes in either receptive or expressive language.
"Beginning in 1998, a worldwide storm of reports on the internet, plus stories
in the Wall Street Journal and on Dateline NBC, have described secretin as
potentially a true treatment for children with autism," said Lightdale. "Our
study and one other were the first to test objectively whether this was so. We
began the study because we were concerned that parents and health care
providers did not have sufficient scientific information to evaluate secretin's
potential effects."
Both studies had negative results. However, the researchers reported that some
parents from both study groups indicated they still would use the drug for
their children.
Lightdale conducted the UCSF study under Heyman's supervision. Their
co-investigators were UCSF-LPPI autism experts Bryna S. Siegel, PhD, Glen R.
Elliott, MD, PhD, Cathy Hayer, MA, and Christopher Lind-White, MD. Lightdale is
now a postdoctoral fellow in pediatric gastroenterology at Children's Hospital
Boston.
Heyman said that parents' excitement about secretin were first stirred by 1998
stories of a mother who said that her son showed improvements in autistic
symptoms after he was given secretin during a diagnostic test for diarrhea.
Dateline NBC profiled the mother and her son. The Wall Street Journal described
an entrepreneur, the father of two autistic children, who founded a
pharmaceutical company and obtained the license for synthetic secretin to
ensure a supply of the drug to treat autism. An article in the Journal of the
Association of Academic Minority Physicians described the first boy, as well as
two other children who reportedly showed increased alertness, eye contact and
expressive language within days after receiving a single dose of the hormone.
The article described marked improvements in language over a short time, and
reported effects lasting up to five weeks.
"After word of these observations spread, UCSF's gastrointestinal service was
inundated with calls from parents asking for secretin infusions, often as many
as 30 per day," Heyman said. Eventually, more than 2,000 called from Northern
California and around the country. "The need for research to see whether the
drug was effective was accentuated by reports from autism interest groups that
thousands of children had begun receiving secretin in repeated doses," Heyman
said. Some web entrepreneurs charged inflated prices to give secretin to
autistic children. In some cases the drug was administered in unproven ways,
such as by mouth or through the skin using a solvent called DMSO.
Siegel, who is associate adjunct professor of psychiatry at UCSF, has worked
with autistic children and their families for 25 years. She defined autism as a
syndrome that begins in the first few years of life, involving severe deficits
in social and communication skills. It affects the child's ability to process
incoming information from the social and physical worlds, to interact with
others and to use language. The cause is not known and, to date, there is no
proven cure. However, for some autistic children, behavioral interventions can
markedly improve language and social skills. For older individuals, medications
sometimes can help ease obsessive behaviors.
Heyman said that secretin is a hormone that stimulates the pancreas to increase
production of digestive fluids. It is approved as a diagnostic drug, to show
whether the pancreas is impaired in its ability to respond to stimulation,
since that can cause chronic diarrhea and other disorders. Secretin has no
known therapeutic use, and few known side effects, though one version of the
drug has been associated with allergic and anaphylactic reactions. It has not
been tested for safety or efficacy in children, either in a single-dose or as a
long-term medication.
Lightdale's study looked at autistic children who were near the same age and
were given a similar intravenous dose of secretin to the three children
described in the 1998 journal article. Each child in the UCSF study was tested
before the infusion with secretin and again in sessions one, two, three and
five weeks after infusion. Each child was given a standardized test called the
Preschool Language Scale - 3, and was videotaped during play and scored for
specific behaviors characteristic of autism.
The PLS-3 test was objectively scored and indicated no quantifiable changes
either in the way children understood language, or the way they were able to
use words and gestures to express themselves. The videotaped behavior tests
were scored using criteria in the test manual, by three independent reviewers
who never met the children and were not told which week of the study a given
test represented. The latter results will be published at a later date.
A true, dramatic change in skills and behavior would have been a surprising
result after a dose of a drug, said Siegel. "The claims made for this drug do
not hold up well to any neuro-developmental model of how new skills are
acquired," she said. "Language ability depends on changes in the brain as the
child goes through activities that stimulate the acquisition of vocabulary and
grammar structure. The child has to develop a two year old's language ability
to go on and learn to speak like a three year old. A pill can't do that for
him."
Eighteen sets of parents in the study filled out a survey afterwards about
their child's condition. In contrast to the results observed by the study
authors, 15 sets of parents indicated that they felt their child had moderate
to significant improvements in language skills following the secretin infusion.
The UCSF results are similar to those of another study, reported in December,
1999 in the New England Journal of Medicine, by researchers at the University
of North Carolina. They studied children aged 3 - 14 who had either autism or a
similar condition, pervasive developmental disorder. They administered secretin
to half and placebo to the rest. The UNC researchers found no significant
effect of secretin - in fact, the children who received the placebo showed
slightly greater improvement on behavior tests. However, a majority of parents,
including those whose children received placebo, retained their interest in
using secretin even after they were told the results.
How to explain the difference between objective test scores and parents'
perceptions? "This is a very good example of placebo effect," said Siegel. "By
definition, the placebo can be expected to cure whatever ails you. In the
studies of secretin, these parents saw differences in eye contact, in
attention, in use of words - any activity that seemed like an improvement was
attributed to the drug."
"Hope is essential when you care for a child with a chronic disability and
there is no definitive treatment," Siegel said. "But sometimes hope lets people
believe more than they truly can count on. It can be a roller-coaster that, in
the end, is just another source of strain."
REPORTERS' NOTES:
* To contact Jenifer Lightdale, call Susan Craig at Children's Hospital Boston
News Service 617-355-6420 craig_s@tch.harvard.edu
* To contact Melvin Heyman or Bryna Siegel, call Janet Basu at UCSF News
Services, 415-476-2557 jbasu@pubaff.ucsf.edu
RE THE PAS/AAP MEETING:
For the first time in their histories, the Pediatric Academic Societies (PAS)
and the American Academy of Pediatrics (AAP) are sponsoring a joint meeting to
discuss the health of children in North America and beyond. Significant
scientific and clinical advances will be presented, touching topics such as
gene therapy, environmental impacts on health, the status of care for neonates,
the mental health of children, and advances in treating chronic diseases.
* Pressroom contacts, May 12-16: Hynes Convention Center, Rm 106, Boston
617-954-2521
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