Philadelphia, PA, 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
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