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New Treatment for Irritability in Autism

ScienceDaily (May 31, 2012) — Autism is a developmental disorder that affects social and communication skills. Irritability is a symptom of autism that can complicate adjustment at home and other settings, and can manifest itself in aggression, tantrums, and self-injurious behavior. These disruptive behaviors are frequently observed in children with autism, which may considerably affect their ability to function at home or in school.

N-acetylcysteine (NAC) is approved by the US Food and Drug Administration for the treatment of acetaminophen (Tylenol) overdoses, but it may have other applications related to its effects in the brain. NAC helps maintain and restore glutathione, which play a key role in the antioxidant defense system. Additionally, cysteine as supplied by NAC treatment, stimulates a protein, the cystine-glutamate antiporter, resulting in the decrease of glutamatergic neurotransmission.

NAC has two resulting effects:


1) it may protect brain cells by raising the level of a protective antioxidant metabolite called glutathione, and


2) it may reduce the excitability of the glutamate system by stimulating inhibitory receptors.

These drug actions are important because, although the causes of autism are unknown, it is clear that there are many influencing factors and scientists are pursuing multiple hypotheses. Two in particular relate to NAC: one theory is that autism may be caused by an imbalance between oxidants and antioxidants in the body; the other is that the glutamate system may be dysfunctional in individuals with autism.

These hypotheses led researchers at Stanford University and the Cleveland Clinic to conduct a pilot trial of NAC in children with autistic disorder. Children were randomized to receive either NAC or placebo daily for 12 weeks and their symptoms were evaluated four times during that period.
They found that irritability was significantly decreased in the children who received NAC. In addition, NAC was well-tolerated and caused minimal side effects....

Pilot Study Finds Promise for Autism in Antioxidant Therapy

By RICK NAUERT PHD Senior News Editor
Reviewed by John M. Grohol, Psy.D. on May 30, 2012

A new pilot study suggests a specific antioxidant supplement may be an effective therapy for some features ofautism.

Researchers from the Stanford University School of Medicine and Lucile Packard Children’s Hospital studied 31 children with the disorder.

The antioxidant, called N-Acetylcysteine, or NAC, effectively reduced irritability in children with autism and moderated repetitive behaviors. The researchers emphasized that the findings must be confirmed in a larger trial before NAC can be recommended for children with autism.

Irritability affects 60 to 70 percent of children with autism. “We’re not talking about mild things: This is throwing, kicking, hitting, the child needing to be restrained,” said Antonio Hardan, M.D., the primary author of the new study.

“It can affect learning, vocational activities and the child’s ability to participate in autism therapies.”

The study will appear in a forthcoming issue of the journal Biological Psychiatry.

Stanford University is filing a patent for the use of NAC in autism, and one of the study authors has a financial stake in a company that makes and sells the NAC used in the trial.

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