Alternative/Complementary

Children with Autistic Spectrum Disorders 33

Approaches to Treatment of Children

with Autistic Spectrum Disorders
Early diagnosis of autistic spectrum disorders (ASD) allows for early referral for treatment and remediation
of core deficits in communication, socialization, and behavior. The cornerstone of treatment is a comprehen-
sive, intensive program of educational, developmental, and behavioral strategies. Since the etiology for most
cases is not well defined, progress may be slow, and treatment may be intense, interest in alternative theories
of causation and novel treatments is high. Families may pursue complementary and alternative medicine
(CAM) therapies in addition to the standard treatments. There are two types of CAM: biologic and nonbiologic.
Some of the treatments have been examined using standard research techniques, while others have not yet
undergone such scrutiny. Families should be supported in their quest for effective treatments and assisted in
learning about potential benefits and harm of each CAM. Key words: autism, autistic spectrum disorders,
complementary and alternative medicine treatments

Susan E. Levy, MD INTRODUCTION

Clinical Associate Professor of Pediatrics
Division Child Development and Rehabilitation
Children’s Seashore House
The Children’s Hospital of Philadelphia
Clinical Associate Professor of Pediatrics
University of Pennsylvania School of Medicine
Philadelphia, Pennsylvania
Susan L. Hyman, MD
Assistant Professor of Pediatrics
Strong Center for Developmental Disabilities
Assistant Professor of Pediatrics
University of Rochester School of Medicine
Rochester, New York
Autistic spectrum disorders (ASD) are diagnosed
with increasing frequency in children under 3 years
of age.1,2 The clinical diagnosis is supported by
identification of core deficits in communication,
social reciprocity, and repetitive behaviors,3 even in
young children.4,5 Although there is much we do
not know about the natural history of symptoms in
preschool children, the diagnosis of ASD is typi-
cally stable in 2- to 3-year-old children.5 The
emphasis on early diagnosis and referral for treat-
ment is based on studies that suggest that intensive
services begun before age 3 might be associated
with better academic and behavioral outcome at
school age.6–8 With increasingly rapid access to
scientific and anecdotal information in the elec-
tronic and print media, families often formulate
opinions about possible interventions that are not
among those conventionally suggested by their
child’s treatment team. A strong belief in a treat-
ment that is considered alternative or complemen-
tary or alternative medicine (CAM) may change the
professional dynamic between professional and
parent. This article reviews some of the common
CAM treatments used to address symptoms of ASD
and discusses strategies that professionals may use
in assessing the validity and safety of new CAM
treatments that emerge.

Inf Young Children 2002; 14(3): 33–42

© 2002 Aspen Publishers, Inc.
33
34 INFANTS AND YOUNG CHILDREN/JANUARY 2002
CONVENTIONAL TREATMENT
RECOMMENDATIONS
Most experts will agree that the best first line of
treatment is an intensive, coordinated program of
special education, developmental therapies, and
behavior management.9,10 A developmentally ap-
propriate intervention program, with a language-
based curriculum for promotion of communication
and social skills, is essential to promote progress.
This program should include intensive speech-
language therapy, with consideration for possible
support with augmentative or total communica-
tion. If the child has sensory intolerance that causes
behavioral dyscontrol, strategies employed by oc-
cupational therapists can be incorporated into the
program to enhance functional abilities and sen-
sory tolerance. Last, the program should incorpo-
rate an intensive behavioral intervention approach
to enhance the acquisition of language and social
behaviors.
Programmatic application of techniques of ap-
plied behavior analysis including discrete trial
instruction has demonstrated improvements in skill
acquisition and function.6,11 Home and educational
programs using DIR (developmental, individual-
difference, relationship-based) approach, also
known as “floor time,” have been advocated for
reinforcement of communication and play skills in
naturalistic settings.12 Other curricula, such as the
TEACCH approach,13 provide an individualized
educational program that is structured to address
the deficits of children with ASD. Although center-
based programs utilizing developmental curricula
have been traditional sources of intervention and
have much anecdotal support, few objective data
have been collected to demonstrate efficacy.9,10
Medication remains a small part of the treatment in
the preschool-age child. For symptoms such as
inattention, hyperactivity, impulsivity, severe ag-
gression, sleep disorders, and others, a trial of
medication may be a helpful adjunct to a behavioral
program14,15 that has been carefully devised to
address these problem behaviors. Medication will
not treat the core symptoms of autism.
A recent policy statement from the American
Academy of Pediatrics16 outlined the role of the
pediatrician in the diagnosis and management of
ASD in children. Therapists and educators are in a
unique position to influence families who may be
pursuing a range of treatments. They may be able
to guide families to seek medical assistance and
avoid serious side effects or complications if they
are cognizant of the basis, benefit, and harm of
popular treatments. The American Academy of
Pediatrics’ recommendations include use of spe-
cific screening tools, referral for audiologic and
speech-language evaluation, and involvement in
early intervention and developmental therapy ser-
vices. Physicians are urged to become familiar with
popular alternative treatments and approach the
issue objectively and compassionately. This is good
advice for educators and therapists as well.
CAM TREATMENTS
It is the job of medical, educational, and other
professionals serving infants and young children to
educate families to review critically promises of
cure or treatment. Families should pursue standard
treatments before considering other ones. Careful
analysis of treatment claims should extend to the
treatments used in conventional intervention pro-
grams as well, since there are often limited outcome
data to justify recommendations for the conven-
tional treatments offered.17 Since developmental
disorders such as ASD have nonspecific treatments
for unclear etiologies, many families seek answers
that provide more hope or definitive promise of
cure.18,19 Families should be taught to become
informed consumers and to review critically the
validity of proposed treatments. They should look
for scientific evidence of benefit, potential health
risk, and financial or time cost of all treatments.
Investigators11 in a study of intensive behavioral
treatments for young children with autism surveyed
 Children with Autistic Spectrum Disorders
families to identify what other approaches they
were employing. Out of 121 families working
closely with treatment teams, 56% had used sen-
sory integration therapies, 50% had tried elimina-
tion diets, and 61% had tried vitamin supplements.
A subset of families was interviewed regarding their
choices of additional therapies. All of those using
diets and vitamins had obtained their information
from other parents or from literature related to ASD.
The original assessment teams were the source of
the recommendation for speech-language therapy.
Therapists outside the treatment team recom-
mended sensory integration.
Since CAM treatment is in such frequent use by
families with children with disabilities, the Ameri-
can Academy of Pediatrics20 recently issued a
position statement. This statement advises pediatri-
cians to be supportive of families in their search for
treatment, careful in their interpretation of claims
from non-peer-reviewed sources, and watchful
over the child, being alert for potential side effects.
Some of the CAM treatments have been sub-
jected to scientific, unbiased study; however, most
claims are based on anecdotal reports or subjective
case series. Young children with ASD may respond
to educational and therapeutic interventions in a
dramatic fashion. The disorder is not static, so
studies of efficacy must be carefully designed to
eliminate confounding factors of treatment and
time effects. Further, the effect of placebo cannot
be discounted.21 To be confident that an interven-
tion is effective, a study must include random
assignment of comparable subjects to different
treatments, measure outcome variables using valid
measures, and employ consistent doses/treatments
across subjects.22
There are two different types of CAM treatments:
biologic and nonbiologic. Biologic agents either
can be bought over the counter by families or
administered by a physician. It is helpful to con-
sider them in three categories as listed in Table 1.
The nonbiologic treatments are novel approaches
to existing therapies; they are provided by a
35
therapist or may be self-administered by the family
or child.
BIOLOGICALS
Vitamin supplements
Several different vitamins and vitamin prepara-
tions are in common use. In most instances they are
not potentially harmful. Many children receive a
combination of vitamin B6 (pyridoxine) and mag-
nesium supplementation, with a high dose of B6 (at
one to two times the Recommended Dietary Allow-
ance). A few controlled scientific studies have
shown some short-term benefits, but most refute
claims of behavioral improvement.23,24 Several stud-
ies have shown some symptomatic benefit, but are
compromised by methodologic problems. A
double-blind, placebo controlled study could not
corroborate the prior findings.24 Potential side ef-
fects include peripheral neuropathy and arrhyth-
mia from magnesium overdose.18
Vitamin C is not as commonly used, but offers
some promise. A small study25 showed decreased
stereotypic behaviors with treatment, but this study
needs to be duplicated. Few side effects are noted.
Dimethylglycine (DMG) was not found to be ben-
eficial in a small case series.26
Vitamin A treatment is based on the assumption
that some children with ASD are vulnerable to the
impact of mercury exposure and that vitamin A
supplementation can change this vulnerability.
This is based on the hypothesis that genetically at-
risk children are predisposed by G-alpha protein
defect. It is assumed by proponents of that theory
that live measles vaccine depletes stores of vitamin
A, resulting in metabolic changes and precipitating
behavior changes in children with ASD.27 Supple-
menting with natural forms of vitamin A (such as
cod liver oil) is purported to improve immune and
visual function. No data are available about effec-
tiveness. There are multiple serious side effects
from overdose, including pseudotumor cerebrii
(increased pressure around the brain). Overdose
36 INFANTS AND YOUNG CHILDREN/JANUARY 2002

Table 1. Summary of complementary and alternative medicine treatments in ASD

Category Type Example

Biologicals
I: Benign; Little basis in “Vitamin” supplement DMG26
theory; Common usage Vitamin supplement B6 and magnesium18,23,24
Anti-infective Antifungal agents41,42
Medication Pepcid18,35

II: Benign; Some basis in Vitamin Vitamin C25

theory, but unproven Diet Gluten-free/casein-free diet48–52
Dietary additive Fatty acids (EFA)55
Medication Secretin28–33
III: Potentially harmful; Medication Chelation (eg, DMSA)56
No basis in theory or Medication Immunoglobulins36–38
unproven scientifically Vitamin Vitamin A (as cod liver oil)34
Anti-infective Antibiotic (eg, vancomycin)
Anti-infective Antiviral agents27,40,41
Medication Alkaline salts18
Medication Bethanecol/urocholine34
Immunization Withhold immunization60
Nonbiologicals
Auditory integration training62
Interactive metronome63
Craniosacral manipulation63
Facilitated communication9,62

DMG, dimethylglycine.

during pregnancy may result in fetal loss and
deformity or damage.
Medications
Secretin is a hormone found in the gastrointesti-
nal tract that helps control digestion. Its use for
children with ASD came to attention after publicity
in 1998 on a national television show. The show
highlighted a case report in the medical literature28
about three children with ASD who improved 5
weeks after secretin administration. These children
had decreased gastrointestinal symptoms and im-
proved behavior. Since then multiple evidence-
based scientific studies have failed to confirm the
claims of dramatic improvement in the symp-
toms.29–33 Despite objective data disproving an asso-
ciation in their individual child, many families who
participated in well-designed, double-blind con-
trolled trials have continued with secretin treat-
ments.21 Potential adverse side effects include im-
munologic or allergic response to repeated admin-
istration of foreign protein and seizures. To date,
there are several hundred children with autism who
have had careful assessment of response to a
double-blind administration of secretin with no
statistically significant benefit. Families who wish
to pursue this therapy may be best served in
controlled trials.
Some families have explored ingestion of alka-
line salts in an effort to provide natural secretion of
secretin and other gastrointestinal peptides.18 Alka-
line salts are potentially harmful to the liver. No
studies of safety or efficacy have been completed,
and there is potential risk in altering the body’s
natural acid-base homeostasis.
Bethanecol is a medication used in gastrointestinal
disorders such as gastroesophageal reflux. Some
clinicians feel it is a pancreatic stimulant and has an
effect similar to secretin. One non-peer-reviewed
source 34 reports that children’s symptoms improved
 Children with Autistic Spectrum Disorders
after treatment with a combination of vitamin A in
cod liver oil and bethanecol. No published results of
controlled studies are available.
Pepcid (famotidine) is an antacid that has been
used because of earlier reports of effectiveness in
treatment of symptoms of schizophrenia.18 One
report35 suggested its use in autism, but no con-
trolled studies have been completed in children
with ASD. There is, however, extensive experience
with famotidine for symptoms of gastroesophageal
reflux in children.
Many investigators have suggested that ASD may
be caused by a dysfunction of the immune system.
There are many reports of abnormalities in the
number and types of antibodies, immunoglobulins,
lymphocytes, and proteins in the central nervous
system.36,37 Laboratory studies showing differences
in measurements of immune markers (eg, elevated
or decreased immunoglobulin levels or antibody
response to viruses) have been used as justification
for treatment with intravenous immunoglobulins
(IVIGs) or antiviral medications. Controlled studies
of IVIG treatment do not document significant
improvement.38,39
Although there are anecdotal reports of children
with ASD treated with antiviral medications typi-
cally reserved for documented systemic viral infec-
tions, there are no data on safety or efficacy. It is
known that prenatal brain damage from rubella
infection during gestation may lead to ASD. It is
plausible that other viruses also might infect the
brain prenatally and cause the types of pathologic
findings frequently seen in the brains of people
with autism.40 However, no such virus has yet been
identified. Immune response to common child-
hood viruses may not be specific for central ner-
vous system infection, and do not imply causal-
ity.27,41 The literature to date does not support the
use of IVIG or antiviral treatments outside of
research protocols.
Antibiotics
It has been hypothesized that in children with
ASD gastrointestinal dysfunction leads to over-
growth of bacteria in the gut. The theory describes
37
possible yeast overgrowth, primary dysfunction of
the immune system, and/or antibiotic overuse. One
treatment includes use of probiotic therapy, where
bacteria living in the gastrointestinal tract are re-
placed with other benign organisms such as acido-
philus (found in yogurt) or treated with antibiotics
such as vancomycin, which eliminate a large num-
ber of gut bacteria. A case report on an Internet
Listserve of several children treated in an open label
trial of vancomycin precipitated great interest in
this treatment. There have been no controlled
scientific studies. Vancomycin has several serious
side effects including colitis-like inflammation and
development of bacteria that are resistant to this
potent antibiotic. In many hospitals, the infectious
disease specialist must be consulted if this antibi-
otic is prescribed in order to regulate the possibility
of creating immune bacteria.
Antifungals
Another theory of causation of autism involves
yeast overgrowth in the gastrointestinal tract due
to excessive treatment with antibiotics. The hy-
pothesis suggests that overgrowth of candida or
yeast, which produces toxins, acts centrally on the
nervous system to produce the symptoms of
autism. The initial evidence for this was circum-
stantial,42 with documentation of substances re-
lated to Kreb’s metabolic cycle intermediates in
the urine of two brothers who developed symp-
toms of ASD with intermittent motor findings.
Proposed treatment of yeast overgrowth would
include lengthy courses of nystatin, Diflucan, and
other medications. Stool cultures may be used as
an endpoint of treatment, but it is not clear if this
is a valid endpoint, as there are no data about how
commonly yeast would be found in large numbers
of children who are asymptomatic. Horvath et al43
did not demonstrate yeast in samples taken from
the small intestines of children with ASD at the
time of endoscopy. These medications have pos-
sible side effects of liver toxicity and anemia.
Dietary means of discouraging yeast overgrowth
have been proposed,44 but have not been exam-
ined for efficacy in clinical trials.
38 INFANTS AND YOUNG CHILDREN/JANUARY 2002
Diet or additives
The idea of dietary treatment of ASD is appealing
to many people. Since many families report gas-
trointestinal symptoms (constipation, diarrhea, and
extreme dietary selectivity), this seems like a logical
source of treatment. In theory, many view it as a
noninvasive, nontoxic means of providing treat-
ment. The theory behind the efficacy of dietary
treatment in ASD, particularly the gluten-free/
casein-free diet, is the opioid excess theory. In
short, this theory states that children with ASD have
impaired function and permeability of the gas-
trointestinal system (“leaky gut syndrome”). Be-
cause of this dysfunction, fragments of wheat
(gluten) or milk (casein) are not well absorbed.
These fragments theoretically function as endog-
enous neuropeptides, or chemical transmitters that
affect brain function.18,45,46 This theory, the common
history of gastrointestinal symptoms, and reports of
effectiveness of gluten-free diet have led investiga-
tors to question whether celiac disease is more
common in children with ASD.47 There are conflict-
ing reports of laboratory studies to confirm gluten
sensitivity in children with ASD. Further controlled
studies of diagnostic evaluations for celiac disease
and impact of gluten-free diet need to be done.
The gluten-free/casein-free diet may be the most
commonly used non-medication treatment at-
tempted, although many families may not imple-
ment the diet correctly with total elimination of
gluten and casein. Anecdotal reports and the lay
press describe remarkable improvement with
implementation of the diet. Since so many children
do have gastrointestinal symptoms, and many
parents report changes in affect and attention, it is
hard to disregard the claims. However, the studies
that have attempted to demonstrate efficacy of this
diet have been flawed. Study problems have in-
cluded lack of valid outcome measures, presence
of confounding treatments, and no placebo con-
trols or challenges.45,46,48 There are two, single-case
methodology reports that were published more
than 20 years ago,49,50 Case series48 have had mul-
tiple confounding factors.
Since information on the diet has appeared
extensively in the media and popular press, many
families undertake this diet without professional
guidance or nutritional support. There may be real
risks in decreased calcium and vitamin D intake in
young children in whom the most abundant source
of these nutrients is cow milk.51 There have been
two recent case reports of children without autism
who were diagnosed as having protein malnutri-
tion after being maintained on rice- or soy-based
beverages by health-conscious parents without
being provided an alternate protein source.52 Pro-
tein sufficiency may be tenuous in children with
ASD given their food preferences, which is further
compromised by additional limitations.53,54 Young
children can have a nutritionally adequate diet that
is milk and wheat free. Nutritional consultation is
indicated, and monitoring by the primary care
provider is essential.
Other approaches have included administration
of essential fatty acids (EFAs) such as flaxseed oil.
The justification for this treatment is extrapolated
from limited data related to children with attention
deficit/hyperactivity disorder (ADHD).56 Supple-
mentation with EFAs is commonly used in an effort
to enhance attention. This has not been assessed
specifically in children with ASD.
Chelation/mercury detoxification
Chelation is one of the more recent treatments
that has reached popularity without adequate sci-
entific scrutiny. Chelation involves the administra-
tion of a chemical (by mouth or through an
intravenous catheter) for the purpose of binding up
and removing from the blood potentially toxic
heavy metals such as iron, mercury, lead, and
others. The theory supporting chelation suggests
that heavy metal intoxication, especially mercury,
is responsible for the regressive form of autism. The
source of mercury is felt to be thimerosal, the
preservative in immunizations. Protocols for chela-
tion treatment in children with ASD have been
proposed and are being implemented by practitio-
ners without benefit of controlled scientific study of
their efficacy for changes in symptoms of ASD.
 Children with Autistic Spectrum Disorders
Recent studies57,58 have not confirmed chelation’s
efficacy in producing changes in developmental
function in children with documented lead intoxi-
cation. The chemicals used for chelation (eg,
DMSA) are not approved by the Food and Drug
Administration for treatment of autism. They are
potentially dangerous, with side effects of liver and
kidney toxicity, potential severe electrolyte and
fluid imbalance, and hypersensitivity.
Immunization controversy
Many families and clinicians believe that mercury
toxicity through exposure in immunizations is a
cause of autism. The belief that symptoms of
mercury toxicity from dietary or industrial sources
might mirror symptoms of ASD has resulted in
concerns about the safety of the use of inorganic
mercury in the form of thimerosal as a stabilizer in
multi-use vials of hepatitis and diphtheria-pertus-
sis-tetanus (DPT) vaccines (see above section on
chelation).
Exposure to organic mercury from maternal
whale meat ingestion in the Faroe Islands has been
associated with language delays in children.59
However, similar doses in the Seychelles from
maternal ingestion of fish did not produce identifi-
able developmental disabilities.60 A record review
of weight and thimerosal exposure in a large cohort
of children in a vaccine monitoring program did not
show an association with ASD.61 A weak associa-
tion with language delays was noted, however.61
Analysis of the existing literature did not support
evidence of harm at the doses of thimerosal found
in vaccines.62 Steps are being taken for removal of
thimerosal from most vaccines. A report evaluating
the putative association of thimerosal and autism
will be published in the near future by the Institute
of Medicine.
Allegations have been made that the measles-
mumps-rubella (MMR) vaccine administration pre-
dated onset of behavioral regression in children
with ASD and gastrointestinal symptoms. 63
Wakefield et al62 theorized that changes seen due to
autistic enterocolitis, including changes in intesti-
nal permeability, were secondary to viral infection
39
from the vaccine invading the gut or perhaps
altering the permeability to potential neurotoxins.
This hypothesis led to a popular belief that admin-
istration of monovalent MMR vaccines, rather than
the trivalent vaccine, would not increase risk of
ASD. Proponents of this theory believe that simul-
taneous administration might lead to persistent
infection or alteration of the clinical and immune
response to the antigens. Review of the scientific
evidence does not support an association at a
population level between ASD and MMR.27,61 There
has been no change in the existing dosage schedule
of childhood vaccines based on these allegations.
NONBIOLOGICALS
A number of noninvasive or nonbiologic treat-
ments also have come into common use. The
popularity of each varies by time and geographic
location, despite a dearth of well-controlled, evi-
dence-based studies to support or refute their
efficacy. The treatments include auditory integra-
tion, facilitated communication, craniosacral ma-
nipulation, and others.
The goal of auditory integration is to decrease
sensitivity to sound by systematic exposure to
altered music by headphones. A well-designed
study described in a recent position paper63 of the
American Academy of Pediatrics did not confirm
positive effects, and the academy does not endorse
this treatment. It is possible that some aspect of the
treatment may be effective for some aspect of ASD
in a subset of individuals. Existing research does
not permit that level of analysis.
The initial positive results of facilitated commu-
nication, the use of a communication device with
physical contact but not conscious guidance from
a familiar person, have not been confirmed when
examined in a blinded fashion. Facilitated commu-
nication was initially proposed in the 1980s. Effects
seen in children have been subsequently shown to
be a function of activities of the facilitator.64
Craniosacral manipulation is a type of manipula-
tion done by chiropractors, physical therapists, and
occupational therapists trained in the technique.
40 INFANTS AND YOUNG CHILDREN/JANUARY 2002
Advocates purport that by massage of the skull they
can alter the flow of cerebrospinal fluid and effect
behavioral change. No scientific treatment data are
available to confirm proposed benefits.9
Interactive metronome is a theoretical treatment. It
involves use of a musical metronome to alter the
timing of information presented to children to en-
hance concentration and vigilance. No studies of
children have been completed. Some preliminary
positive results were reported at a conference, based
on some results seen in children with ADHD.64
SUMMARY
There are many factors that contribute to parents’
choices of treatment for children with ASD. Some
of those choices may not include or may interfere
with traditional early intervention services. All
professionals involved in the therapeutic manage-
ment of children with ASD need to be aware of all
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