CM E

Scientifically Unsupported Therapies

in the Treatment of Young Children with
Autism Spectrum Disorders
Merryl A. Schechtman, MD

A
utism spectrum disorders
(ASD) are among the most
puzzling forms of develop-
mental disability. This term refers to
a spectrum of pervasive developmen-
tal disorders sharing deficits in three
major domains: social relatedness and
interactivity, communication, and re-
stricted interests and/or stereotypic or
repetitive behaviors with difficult tran-
sitions. Studies have investigated ge-
netic factors, neurologic, immunologic,

CM E EDUCATIONAL OBJECTIVES

1. Identify the domains of comple-

mentary and alternative therapy.
2. Describe the basis for the brain-gut
connection hypothesis in autism
spectrum disorders.
3. Discuss sensory issues in children
with autism and sensory integra-
tion therapy.

Merryl A. Schechtman, MD, is Assistant

Clinical Professor of Pediatrics, Albert Ein-
stein College of Medicine, Infant Preschool
Unit, Childrens Evaluation and Rehabili-
tation Center, Rose F. Kennedy Center.
Address correspondence to: Merryl A.
Schechtman, MD, 1410 Pelham Parkway
South, Bronx, New York 10461; fax 718-
892-2296.
2007/Jupiter Images/Corbis

Dr. Schechtman has disclosed no rel-

evant financial relationships.

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 and early environmental TABLE. The prevalence of com-
insults. ASD are lifelong, plementary and alternative
often severe disorders Types of Complementary therapy usage in children
impacting all facets of and Alternative Medicine with chronic illness, de-
individual, family, and Domain Examples velopmental disabilities,
community function. Re- Alternative medicine systems Chinese medicine, acupuncture and emotional problems
lationships are disrupted, Mind-body interventions Meditation
is high.1-3 These treat-
and socially inappropri- ments are often promoted
Biologically-based medicine Megavitamins, elimination diets
ate behaviors are promi- commercially. They also
nent. Often these children Body-based therapies Sensory integration therapy generally lack objective,
do not cope well with Energy therapies Magnet therapy evidence-based studies to
change, making it diffi- support their claims of ef-
cult for families to go out
in public places. Children may become
SIDEBAR.
aggressive, self-injurious, or resort to
self-stimulatory behaviors, which serve Biologically-based Therapies
to calm them. Although the underlying
Elimination diets: Feingold diet, gluten- and casein-free diet, ketogenic diet
causes of ASD have not been clarified,
Vitamins and supplements: Vitamins A, B6, B12, C, folate, magnesium, dimethylglycine,
several promising interventions have omega 3 fatty acids
been developed utilizing behavioral Neurosecretory agents: Secretin, oxytocin
techniques, educational programs, and Famotidine (Pepcid)
pharmacotherapy to address the symp- Immunologically-based therapies:
toms of this disorder. The prognosis for Antibiotics: vancomycin
truly normal function is guarded, even Antivirals
with availability of standard therapies, Antifungals based on the theory of yeast overgrowth (nystatin, fluconazole)
a factor often leading to parental des- Intravenous immunoglobulin G
peration and the willingness to invest Toxin removal: Chelation therapy based on theory of mercury toxicity
in newer approaches to treatment. Ef- Non-biologically based treatments: Sensory integration therapy, facilitated
communication therapy, auditory integration therapy, hyperbaric oxygen therapy
fective evidence-based services may
also not be available or in short supply.
Standard therapies may require much
time to see progress, and therefore modern-day medicine has been treat- ficacy, often relying upon unproven the-
some parents may be willing to grasp at ment that is evidence-based. There has ories with results that are usually based
the promise of a quick fix. been a growing trend toward the use of upon anecdotal cases. Commercial Web
Scientifically unsupported therapies scientifically unsupported approaches, sites or publications, instead of peer-re-
lack a foundation based on objective, especially within the fields of chronic viewed, scientific journals, are the ve-
controlled research, using methods illness and developmental disabilities. hicles promoting the information to the
standardized within biological or medi- In 1992, the U.S. government estab- public. Therapies reaching the attention
cal science. Therapies such as these are lished the National Center for Comple- of the media may then be sensational-
often used either in conjunction with mentary and Alternative Medicine (NC- ized, furthering their promotion. Often
evidence-based medical treatments CAM) through the National Institutes of pediatricians find themselves in a difficult
(complementary therapies) or might be Health. The NCCAM recognizes five do- position imposed by a parent requesting
used instead of the standard medical mains of complementary and alternative their endorsement of an unsubstantiated
practice (alternative therapies). Use of medicine: alternative medicine systems treatment.4 Recent studies found the use
these therapies date back to early folk (eg, Chinese medicine), mind-body inter- of CAM to range between 50% to 92% in
medicine and are often based upon be- ventions (eg, meditation), biologically- the ASD population.5,6 Within the ASD
lief systems stemming from religious based medicine (eg, megavitamin ther- population, 50% of biologically-based
faith. Although the use of non-stan- apy), body-based therapies (eg, sensory therapies utilized by parents involved
dardized home remedies for minor ill- integration therapy), and energy therapies dietary changes.7 Seventy-six percent of
nesses is widespread, the emphasis in (eg, magnet therapy) (see Table). parents using dietary modifications felt

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 that these had been of some benefit for al reflux disorder, which may have been Erickson11 also reviewed the use of
their affected child. In the category of a factor in their improvement. the ketogenic diet in a small population
body-based therapies, chiropractic ther- Dietary manipulation in the treat- of children with autism. Although im-
apy was most commonly used; however, ment of developmental or behavioral proved behavior was noted, the study
there was no real reported benefit. Vari- problems is not a new concept. The ori- was not controlled. Dietary limitations,
ous therapies were reported by parents gin of this practice is beyond the scope such as those imposed by selective
as helpful in relaxing the child diagnosed of this article. However, more recent elimination diets, may impose addition-
with ASD, and these included therapeu- dietary practices based upon presumed al stress on children and their families
tic horseback therapy (hippotherapy), associations with nutritional excesses and hypothetically may result in nutri-
massage, music therapy, and sensory in- and deficiencies and possible effects tional deficiencies.
tegration therapy. on behavior evolved in the 1920s, with In 1998, Horvaths report on im-
This article will focus on many of the suggestion of excessive sugar con- proved developmental/behavioral func-
the biologically and non-biologically sumption and the development of hy- tion following secretin administration
based alternative or complementary peractivity and impulsivity. The Fein- to children with gastrointestinal com-
therapies used in the treatment of ASD gold diet,12 which became popularized plaints and symptoms of autism added
(see Sidebar, page 640). in the 1970s, was largely based upon to the growing number of theories of
these theories. brain-gut interaction based upon gas-
BIOLOGICALLY-BASED THERAPIES In the 1970s, Panskepp13 proposed trointestinal proteins acting as central
that malabsorption of casein (a milk pro- neuropeptides in the brain.15 However,
Diets: The Brain-Gut Connection tein) and gluten (a wheat protein) might despite the initial excitement of se-
The potential relationship between be responsible for manifestations of au- cretin as a treatment in ASD, multiple
gastrointestinal symptoms and autism tism via altered cerebral neurotransmit- well-controlled, peer-reviewed stud-
has been the basis of much investigation ter metabolism. The protein metabolites ies on more than 700 children did not
in this population. Valicenti-McDermott from milk and wheat products were bear out the treatment effects initially
et al8 reported a higher than expected postulated to be absorbed through a reported by Horvath.
frequency of gastrointestinal leaky gut and to act central- In 2002, Wakefield16 reported a case
symptoms and food selec- ly as endogenous opioids; series of endoscopic studies on children
tivity in a sample of 50
children with ASD.
Increased symptoms
such as diarrhea, re-
The U.S. Institute of Medicine report ...
flux, constipation, concluded that there is no evidence linking the
bloating, etc., have
been reported with
MMR vaccine with autism.
frequencies of 9%
to 50% in this popu-
lation.9 Increased lev-
els of serotonin, a primary however, the relationship with autism and gastrointestinal symp-
gut neurotransmitter, have been between opioid activity and the toms. An increased rate of ileal lym-
found in the plasma of people with au- development of autism remains specula- phonodular hyperplasia and colitis was
tism, but it is not clear that this is linked tive. The finding of increased peptides reported. This article has since been re-
to the gastrointestinal symptoms.10 in the urine of children with autism14 is tracted by almost all of the authors.
In a small, double-blinded, placebo- controversial, as is the proposal that a
controlled study reviewed by Erickson,11 gluten- and casein-free diet can decrease The Immune System Connection
famotidine (Pepcid) was administered urinary peptides, resulting in increased Abnormal immune function in chil-
to children with ASD and gastrointesti- social interactivity among autistic chil- dren with ASDs has included the find-
nal symptoms. Improved behavior was dren. Despite the lack of definitive data, ings of increased antibodies, abnormal
noted; however, four of the nine chil- the use of the gluten-/casein-free diet is cytokines, antibodies to myelin basic
dren noted to have behavioral improve- widespread with many anecdotal testi- protein, abnormal immune responses,
ment had undiagnosed gastroesophage- monials of its success. and a reported increased prevalence of

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 lymphonodular hyperplasia in a previ- co-authors published a formal retrac- agent be used for diseases where there
ously diagnosed population of children tion of the suggestion of a link between was documented benefit.
with ASD.16-18 Despite suspicion of im- MMR and autism in The Lancet.24 The
mune or autoimmune dysfunction in U.S. Institute of Medicine report co- IS IT HEAVY METAL TOXICITY?
children with ASD based upon reports of sponsored by the National Institutes of Bernard et al28 drew attention to sim-
increased gastrointestinal symptoms, ear Health and the Centers for Disease Con- ilarities between symptoms of autism
infections, and allergies, no increase in trol and Prevention concluded that there and mercury toxicity, suggesting a con-
the rate of either ear infections or allergic is no evidence linking the MMR vaccine nection between environmental sourc-
responses, has been noted.19,20 However, a with autism.25 es of mercury and the development of
recent report revealed a higher family his- autism. However, mercury poisoning is
tory of autoimmune disorders (rheuma- IS IT BACTERIAL? characterized by severe movement dis-
toid arthritis, celiac disease, and inflam- Sandler et al26 used the antibiotic van- orders and peripheral nerve damage,
matory bowel disease) in a population of comycin in a small study of 11 children none of which is typically found in
children with ASD and gastrointestinal with autism and diarrhea whose stools children with ASD. Nonetheless, me-
symptoms who had undergone language were colonized with a different clostridi- dia reports led to requests for mercury
regression, compared with those without um species than controls. It was suggested testing because of growing concerns
language regression.21 that neurological symptoms might result of possible toxicity. Mercury, a heavy
from altered gut integrity on the basis of metal found in the environment, accu-
IS IT VIRAL? toxins in altered colonic flora. However, mulates in internal organs. Therefore,
Levy and Hymans 2005 review22 cited outcome parameters of this study were blood and urine assays do not always
investigational reports of evidence of early not totally blinded, and the childrens be- reveal mercury exposure. Hair trace
infection, chronic inflammation, or au- haviors may have improved because of analysis, which has not been standard-
toimmune disorders as possible etiologic improved bowel function alone. ized, is not considered to be a reliable
factors in ASD. They commented that assay for mercury toxicity because of
prenatal and neonatal exposure to viruses IS IT FUNGAL? differing rates of hair growth, variable
is postulated to alter brain development; In the 1980s, anecdotal reports impli- hair composition, and because sham-
however, studies of viral exposure failed cated overgrowth of the yeast Candida poos, hair products, exposure to sun,
to show an inflammatory response in the albicans in precipitating some cases and drying all serve to leach substances
brain. Antiviral agents have been proposed of autism. Crook27 suggested the over- from hair. Thimerosal, an ethyl-mer-
as a treatment strategy; however, no spe- growth of yeast to be secondary to anti- cury based preservative once used in
cific virus has been identified, and no pub- biotic use or ingestion of processed sug- many vaccines in the United States (in-
lication has addressed antiviral therapies ars. Candidal overgrowth was, however, cluding the MMR vaccine), was sug-
in terms of efficacy or safety in ASD. not documented by endoscopic study.16 gested as a possible cause of autism
In 1998, Wakefield16 reported on 12 The yeast theory popularized the use of in a subpopulation of immunized chil-
cases of children with GI abnormalities treatments to reduce yeast colonization, dren. Other sources of mercury such as
and lymphonodular hyperplasia, and which included the use of probiotics (ac- that contained in thermometers, mer-
eight of the 12 children allegedly dem- idophilus, lactobacillus), dietary modifi- cury amalgams in tooth fillings, and
onstrated symptoms of autism reported cation (reduction of refined sugars), and some fish came under scrutiny in the
to develop soon after receipt of an MMR the use of antifungal agents like nystatin search for environmental precipitants
vaccination. Although Wakefield did and fluconazole. of autism symptoms. Pregnant women
not prove an actual causal connection were issued warnings against exces-
between autism and MMR, public con- IS IT PRIMARY sive fish consumption, and amalgam
cern nonetheless exploded after this was IMMUNODEFICIENCY? removal was even suggested. Madsen
reported on 60 Minutes. Madsen et al23 Levy and Hyman22 reviewed three et al23 examined the use of thimerosal-
using national-registry data on autistic small case series of ASD with equivo- containing vaccines and ASD in Den-
disorders in Denmark (a national cohort cal results after intravenous immu- mark, where thimerosal was removed
study including 537,000 children) found noglobulin G (IVIG) administration. from vaccines in 1992. This study
no association between the MMR vac- However, because of the small risk of demonstrated that despite removal of
cine and the subsequent diagnosis of au- blood-borne infection and side effects thimerosal from vaccines, the numbers
tism. In March 2004, 10 of Wakefields of IVIG, they recommended that this of autism cases continued to rise at

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 the same rate as prior to its removal. studies at that time, many of which were environmental contaminants. This study
Studies of children who received heavy not blinded or adequately controlled. In suggested that as plasma concentrations
metal chelation failed to demonstrate a review of the literature in 2002, Nye of the antioxidants increased, behaviors
improved neurodevelopmental func- and Brice34 concluded that no recom- were noted to improve; however, further
tion on follow-up.29 More ominous mendation could be made for this treat- studies have not replicated this data.
was the documentation of two deaths ment based upon the scant data available. Dimethylglycine (DMG, a nutritional
in children in the United States (one of Caution is advised because excessive in- supplement) is metabolized to the excit-
whom had autism) because of take of pyridoxine is associated atory neurotransmitter, glycine, within the
hypocalcemia and cardiac with peripheral neuropathy. liver. There have been reports of an old
arrest after undergo-
ing chelation thera-
py.30 The American Although there is no evidence for mercurys
Academy of Pedi-
atrics (AAP) and
role in causing ASD, chelation therapy has been
the Public Health utilized as a detoxification procedure, with
Service published
a statement in 2001 occasionally disastrous results.
indicating the lack of
a decrease in the prev-
alence of autism despite Excessive magnesium Russian study, which suggested enhanced
the removal of thimerosal.31 concentrations are associated language skills in developmentally disabled
Although there is no evidence for with the development of seizures. children who were administered DMG;
mercurys role in causing ASD, chela- Vitamin A, such as that found in however, two well-controlled and blinded
tion therapy has been utilized as a de- cod liver oil, is promoted as improv- studies in ASD failed to demonstrate a dif-
toxification procedure, with occasion- ing immune function and vision in ference between DMG and placebo.37,38
ally disastrous results. children with autism; however, no Omega-3 fatty acids or polyunsatu-
data are available. Excessive use of rated fatty acid (PUFA) 39 is a popular
VITAMINS AND NUTRITIONAL Vitamin A can lead to hepatotoxicity treatment for a variety of developmental
SUPPLEMENTS and increased intracranial pressure. problems including attention-deficit/hy-
Nutritional supplements have also been Vitamin C (ascorbic acid) was peractivity disorder, dyslexia, develop-
popularized in the treatment of symptoms reported in one trial to decrease ste- mental coordination disorder, and ASD.
in children with ASD. Megavitamin ther- reotypic behaviors significantly in a The results of controlled treatment trials
apy gained popularity in the 1960s with study of residential students with au- have been mixed and are hard to inter-
Linus Paulings theory linking mental ill- tism; however, this study was never pret because of differing formulations
ness and inborn errors of metabolism.32 A replicated.35 Vitamin C in excessive of the essential fatty acids and different
variety of vitamins (vitamins A, C, B6- doses has the potential to cause diar- populations treated. Larger clinical trials
magnesium complex, folic acid, B12) and rhea and renal stones. are needed to corroborate the findings of
minerals have been advocated. Vitamin B12 treatment was recom- a few reports of improved behaviors.
Vitamin B6-magnesium complex has mended based upon data from a small, Oxytocin intranasally and intrave-
been the most heavily promoted of the controlled study of 20 children with nously may have significant positive
vitamin therapies for ASD. Vitamin B6 autism found to have lower plasma con- effects on repetitive behaviors and so-
(pyridoxine) has been advocated because centrations of the antioxidants methio- cial cognition in adult autism patients;
of its cofactor role in the production of nine, homocysteine, total glutathione, however, the significance of these re-
the neurotransmitters: serotonin, norepi- cysteine, and S-adenosylmethionine as sults needs further investigation as
nephrine, epinephrine, dopamine, and compared with controls.36 Administra- studies on adults cannot be general-
GABA. Magnesium may have an addi- tion of subcutaneous vitamin B12 and ized to children.40
tive effect on this. Rimland33 reported oral folinic acid to patients with ASD
decreased behavioral outbursts in autism is based on the premise that these indi- Other Supplements
with vitamin B6-magnesium supple- viduals have compromised antioxidant Levy and Hyman reviewed data on
mentation, based on more than a dozen defenses with an inability to detoxify other supplements such as tryptophan,

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 tyrosine, cyproheptadine, D-cycloser- puter or typewriting device in which an- brain injury, and ischemic brain injury.
ine, and carnosine. There is need for other individual, the facilitator, guides the It was postulated that decreased blood
additional research to support the use hand of a nonverbal individual. This tech- perfusion to several areas of the brain,
of these supplements.22 nique was originally used to assist physi- in particular the temporal regions and
cally disabled individuals.42 Numerous auditory processing and language areas,
NONBIOLOGICAL INTERVENTIONS controlled and blinded studies have failed correlate with many of the behaviors as-
to demonstrate FC as replicable or valid. sociated in autism.47 However, scientific
Sensory Integration Therapy In 1998, the AAP issued a statement from evidence is lacking for the use of HBOT
Ayres, an occupational therapist, pos- the Committee on Children with Disabili- in developmental disabilities.
tulated in 1979 that children with ASD ties highlighting the lack of scientific data
have deficits in the brain areas responsi- to show FC to be effective.43 SUMMARY
ble for processing sensory input (visual, Our understanding of ASD has
tactile, auditory, gustatory, vestibular, Auditory Integration Training changed over the past decades, and di-
and proprioceptive) and motor output.41 Auditory integration training (AIT) agnostic tools have assisted in earlier
Sensory integration activities include is a technique conceived by a French identification and referral for interven-
jumping on trampolines, swinging, spin- ENT specialist, Dr. Guy Berard, in tion. Appropriate intervention appears
ning the body, rolling the body, riding a the 1960s. It consists of acoustically to impact positively on overall outcome
scooter board, balance activities, appli- modified music played to a child for 10 for a pervasive developmental disorder
cation of brushes to the body, the wear- hours in two 30-minute sessions each for which there is currently no known
ing of weighted vests, smooshing a day from a CD player attached to box cure. Novel and controversial thera-
child between pads or pillows, and play- (AIT device) that is wired to modify the pies will come and go, and therefore
ing with textured toys. Oral motor activ- signal, presumably to reduce the vol- physicians should familiarize them-
ities may be used as well. Manipulation ume for frequencies to which the child selves with these interventions, as ad-
of the environment is central to sensory is hypersensitive. It was publicized as a vice about these alternative approaches
integration therapy; therefore, the fabric method to retrain the auditory system will be sought. Discussions of nontra-
(texture) of clothing or sheets may be in children with auditory sensitivities ditional therapies should include the
changed. Labels and tags are removed and became popularized by the book placebo effect, possibly undesirable,
from clothing, and class sizes are limited The Sound of a Miracle by Stehli.44 or potentially dangerous outcomes of a
to decrease distraction. Within the class- Gravel45 stated that there was no scien- treatment, and the importance of scien-
room, a quiet corner is established to pro- tific evidence for the type of peripheral tifically sound research studies of that
vide an area with decreased stimulation. hearing abnormalities that Berard orig- treatment. Addressing the use of com-
Occupational therapists commonly pro- inally reported. In addition, the sound plementary and alternative therapies in
vide sensory integration therapy, which pressure levels produced by some of the families with medically compromised
may take place in differing venues, such AIT devices were potentially unsafe. or developmentally disabled children
as in the home or school. Anecdotal evi- The 1998 statement of the AAP, which is crucial to providing complete care to
dence for efficacy of sensory integration addressed Facilitated Communication,42 the patient.
therapy is widespread, and there is great also indicated the lack of scientific evi- A note from the editors:
interest in establishing an evidence-base dence and potential danger of Auditory This article originally appeared in
for these therapies. Integration Training, recommending its Pediatric Annals, a SLACK Incorporat-
There is ongoing research evaluating use in experimental protocols only. ed publication.
the use of water therapy (aquatherapy),
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