Effectiveness of Over-the-counter Therapies for Autism

Background: Many parents of children with autism provide their children with over-the-counter (OTC) therapies for autism often based on recommendations by complementary and functional medicine practitioners.  Existing medical literature is available on many of these therapies.

Objectives: Identify therapies for autism that have been examined in the literature and determine which have the best risk-adjusted efficacy.

Methods: A literature search was conducted to identify OTC therapies for autism.  Each therapy was then evaluated for biochemical rationale, efficacy, possible side-effects, risk, possible indications, and possible contraindications.  A composite assessment was also made for each therapy taking these factors into account.

Results: Thirteen therapies were identified in the literature.  Three of the therapies rated “Excellent” on a risk-adjusted composite assessment: methylcobalamin & folinic acid, omega-3 fatty acids, and probiotics.  Three of the therapies rated “Good” on a risk-adjusted composite assessment: carnitine, CoQ10, and NAC.  Some of the therapies that were shown to have modest or excellent efficacy did not score as well on the composite assessment due to risk, side-effects or other issues.  Two therapies were shown to have no efficacy based on placebo controlled trials: pyridoxine & magnesium and inositol.

One may object that the methylcobalamin & folinic acid trial run by James included methylcobalamin as injections, which are not available over-the-counter.  However, the literature and anecdotal evidence suggests that methycobalamin is absorbed orally even in those with digestive conditions.  Therefore this therapy may be applied orally where it is available over-the-counter.

Three of the therapies with significant promise based on efficacy alone are sulfur containing compounds: NAC, DMSA, and sulforaphane.  This likely is not coincidental given that high sulfur excretion and low cysteine levels are common in autism.  These therapies did not score as well on the composite measure as all three may have significant side effects based on current literature.  This suggests a potential area of research in autism: identification of sulfur containing compounds that are efficacious with negligible side-effects.

Some therapies that are widely used did not score well on a composite basis.  These therapies include pyridoxine & magnesium, multivitamins, and vitamin D.  Pyridoxine & magnesium had poor efficacy.  Multivitamins and vitamin D were both determined to have a mixed biochemical rationale: literature suggests that excessive levels of monoamine neurotransmitters, sulfation deficits, and excessive growth in infancy are common in autism.  As multivitamins and vitamin D both tend to exacerbate these conditions, they are mixed on this measure of rationale.

There are known or inferred indications and contraindications with most of the therapies identified.  Therefore in the hands of a careful and educated practitioner who is willing to run preliminary tests of biochemistry such as organic acid tests, it is possible to further mitigate risk by selecting therapies that appear to fit with an individual’s biochemistry.

Conclusions: The literature suggests three of the OTC therapies have excellent risk-adjusted efficacy and three have good risk adjusted efficacy.  Practitioners have a number of OTC therapy options currently available for treatment.  They should take advantage of them.