CAM Treatments for Use in Children and Adolescents with Autism

Complementary and alternative medicine (CAM) is used by many children and adolescents with autism spectrum disorders. The National Center for Complementary and Alternative Medicine, a branch of the National Institutes of Health, described CAM as “a group of diverse medical and health care systems, practices, and products that are not generally considered part of conventional medicine.” What is Complementary and Alternative Medicine? (NCCAM/NIH)
It is important to ask patients and their parents if they are taking CAM treatments. Rates of CAM usage in children with ASDs are quite high, ranging from 52-95% in some reports. [Akins: 2010] Clinicians at The M.I.N.D. Institute at the University of California-Davis have published a comprehensive review article reviewing the evidence behind may CAM treatments and have created a useful classification system evaluating many of these treatment strategies.
The following classifications have been suggested to categorize CAM therapies based on efficacy, tolerability, and safety. For a complete description regarding the following treatment ratings and recommendations. [Akins: 2010] [Anagnostou: 2011]

Well-tolerated treatments showing efficacy

Recommend use as a first line treatment
Melatonin - This medication is well-tolerated and has been shown to help improve sleep patterns in autism spectrum disorders. Continue to encourage behavioral management and good sleep hygiene. [Rossignol: 2011] [Andersen: 2008]
Multivitamins - Safe at recommended doses. Since children with ASDs may have very strict, particular diets, supplementation with a multivitamin can help with possible nutritional deficiencies. It is important to avoid giving over the recommended daily amount to avoid vitamin toxicity.

Well-tolerated treatments with inconclusive/unknown efficacy

Support use if a family indicates desire to try, but encourage objective monitoring of the treatment
*Vitamin C - Safe at recommended doses and from diet. Some feel it has a role in oxidative stress, but there are no high quality studies examining the use of Vitamin C in ASDs.
Gluten/casein free diet - The theory behind this diet regimen is that some children with ASDs are unable to break down the proteins in gluten and casein in their food, leading to formation of opioid proteins that act on the brain to cause some of the symptoms of autism. The current evidence is poor, including mostly case reports and small studies, but this diet regimen is still being studied. It is important to counsel families using this diet about the importance of adequate calcium, protein, and Vitamin D intake since bone loss has occurred in some children on this diet regimen. [Millward: 2008] [Hediger: 2008]
*Vitamin B6, magnesium - Considered a safe treatment with inconclusive efficacy based on studies. A recent Cochrane review did not show significant evidence to support its use in children with autism. [Nye: 2005] The Institute of Medicine has recommended maximum intake of vitamin B6 of 100mg per day in adults. [Akins: 2010]
Amino acids (Carnosine) - This is an amino acid felt to have antioxidant properties that is relatively safe, but efficacy was only shown in one controlled trial over 8 weeks. Adverse effects can include irritability and hyperactive symptoms. In one study of 31 children with ASDs, 800mg of L-carnosine was given with improvement on receptive language scores. These results have not repeated though. [Chez: 2002]
*Essential fatty acids (omega 3 fatty acid) - Though appear to safe treatments, their efficacy is inconclusive. Omega-3 fatty acids are found in fish and fish oil supplements. A recent Cochrane review demonstrated the lack of good quality studies to support its efficacy in this population. [James: 2011]
*Methyl B12 (methylcobalamin), folate, & glutathione - Considered safe treatments, but efficacy inconclusive.
Music therapy - A recent Cochrane review of music therapy in ASD noted some improvements in gestures and verbal abilities in a few smaller studies. [Gold: 2006]
Acupuncture [Cheuk: 2011]
*If a child has a measured nutritional deficiency, replacement therapy with the appropriate nutrient would be considered standard treatment and one should follow established guidelines.

Well-tolerated treatment, but no evidence of efficacy

Discourage use
Secretin - Secretin is a peptide in the GI tract that is used to treat peptic ulcers and evaluate pancreatic function. It was felt from animal studies that secretin could act as a neurotransmitter. It has been studied in may RCTs and has shown no evidence for effectiveness. [Krishnaswami: 2011] [Williams: 2012]

Unsafe or unknown safety and inconclusive or no efficacy

Discourage use
Chelation therapy - Chelation therapy is unsafe and should be discouraged as deaths have been reported in children. This treatment also has not demonstrated efficacy. Chelation therapy was created to treat lead poisoning, but was marketed as a treatment for ASD patients by stating they have problems eliminating heavy metals from the body. There are also potential serious side effects with treatment such as kidney and liver problems, neutropenia, neurologic problems, and Stevens-Johnson syndrome.
Hyperbaric oxygen therapy - This treatment has no evidence for efficacy in studies and there may be safety concerns related to these treatments. This therapy has been very effective for treatment of decompression sickness, carbon monoxide poisoning, or wound infections. These treatments can cost thousands of dollars, are time consuming, and can be associated with exacerbation of seizures and pulmonary issues.
Immune therapies - Safety and efficacy for these therapies is unknown and IVIG treatments can have severe SE including systemic reactions to the infusion.
Antifungal agents - These treatments are unsafe and efficacy has not been demonstrated in studies to treat behavioral issues or core symptoms of autism.


Information & Support

For Professionals

Genetics in Primary Care Institute (AAP)
The goal of this site is to increase collaboration in the care of children with known or suspected genetic disorders. Includes health supervision guidelines and other useful resources; a collaboration among the Health Resources & Services Administration, the Maternal and Child Health Bureau, and the American Academy of Pediatrics.


Authors: Tara Buck, MD - 6/2012
Catherine Jolma, MD - 8/2009
Deborah Bilder, MD - 8/2009

Page Bibliography

Akins RS, Angkustsiri K, Hansen RL.
Complementary and alternative medicine in autism: an evidence-based approach to negotiating safe and efficacious interventions with families.
Neurotherapeutics. 2010;7(3):307-19. PubMed abstract / Full Text
This review focuses on helping clinicians identify resources and develop strategies they may use to effectively negotiate safe and effective use of complementary and alternative medicine (CAM) treatments with families of children with autism spectrum disorders (ASD), as well as other neurodevelopmental disorders.

Anagnostou E, Hansen R.
Medical treatment overview: traditional and novel psycho-pharmacological and complementary and alternative medications.
Curr Opin Pediatr. . 2011;23(6):621-7. PubMed abstract
Accumulating data suggest a series of existing medications may be useful in ASD and large randomized clinical trials are necessary to evaluate safety and efficacy of both pharmaceuticals and alternative treatments.

Andersen IM, Kaczmarska J, McGrew SG, Malow BA.
Melatonin for insomnia in children with autism spectrum disorders.
J Child Neurol. 2008;23(5):482-5. PubMed abstract

Cheuk DK, Wong V, Chen WX.
Acupuncture for autism spectrum disorders (ASD).
Cochrane Database Syst Rev. 2011;(9):CD007849. PubMed abstract / Full Text
To determine the effectiveness of acupuncture for people with ASD in improving core autistic features, as well as communication, cognition, overall functioning and quality of life, and to establish if it has any adverse effects.

Chez MG, Buchanan CP, Aimonovitch MC, Becker M, Schaefer K, Black C, Komen J.
Double-blind, placebo-controlled study of L-carnosine supplementation in children with autistic spectrum disorders.
J Child Neurol. 2002;17(11):833-7. PubMed abstract / Full Text
L-Carnosine, a dipeptide, can enhance frontal lobe function or be neuroprotective. It can also correlate with gamma-aminobutyric acid (GABA)-homocarnosine interaction, with possible anticonvulsive effects. Although the mechanism of action of L-carnosine is not well understood, it may enhance neurologic function, perhaps in the enterorhinal or temporal cortex.

Gold C, Wigram T, Elefant C.
Music therapy for autistic spectrum disorder.
Cochrane Database Syst Rev. 2006;(2):CD004381. PubMed abstract / Full Text
Music therapy uses music and its elements to enable communication and expression, thus attempting to address some of the core problems of people with ASD. The findings indicate that music therapy may help children with autistic spectrum disorder to improve their communicative skills. More research is needed to examine whether the effects of music therapy are enduring.

Hediger ML, England LJ, Molloy CA, Yu KF, Manning-Courtney P, Mills JL.
Reduced bone cortical thickness in boys with autism or autism spectrum disorder.
J Autism Dev Disord. 2008;38(5):848-56. PubMed abstract

James S, Montgomery P, Williams K.
Omega-3 fatty acids supplementation for autism spectrum disorders (ASD).
Cochrane Database Syst Rev. 2011;(11):CD007992. PubMed abstract / Full Text
Reviewed the efficacy of omega-3 fatty acids for improving core features of ASD and associated symptoms. To date there is no high quality evidence that omega-3 fatty acids supplementation is effective for improving core and associated symptoms of ASD.

Krishnaswami S, McPheeters ML, Veenstra-Vanderweele J.
A systematic review of secretin for children with autism spectrum disorders.
Pediatrics. 2011;127(5):e1322-5. PubMed abstract / Full Text
Secretin has been studied extensively in multiple randomized controlled trials, and there is clear evidence that it lacks benefit. The studies of secretin included in this review uniformly point to a lack of significant impact of secretin in the treatment of ASD symptoms.

Millward C, Ferriter M, Calver S, Connell-Jones G.
Gluten- and casein-free diets for autistic spectrum disorder.
Cochrane Database Syst Rev. 2008;(2):CD003498. PubMed abstract / Full Text

Nye C, Brice A.
Combined vitamin B6-magnesium treatment in autism spectrum disorder.
Cochrane Database Syst Rev. 2005;(4):CD003497. PubMed abstract
Due to the small number of studies, the methodological quality of studies, and small sample sizes, no recommendation can be advanced regarding the use of B6-Mg as a treatment for autism.

Rossignol DA, Frye RE.
Melatonin in autism spectrum disorders: a systematic review and meta-analysis.
Dev Med Child Neurol. 2011;53(9):783-92. PubMed abstract / Full Text
Melatonin administration in ASD is associated with improved sleep parameters, better daytime behavior, and minimal side effects.

Williams K, Wray JA, Wheeler DM.
Intravenous secretin for autism spectrum disorders (ASD).
Cochrane Database Syst Rev. 2012;4:CD003495. PubMed abstract / Full Text
Over 900 children were recruited for the secretin trials. Meta-analysis of data was not possible but there is now consistency of findings, with RCTs of the efficacy of secretin in autism not showing improvements for core features of ASD.