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New autism treatment shows promising results in pilot study

An antioxidant supplement has shown early promise as a potential autism therapy, according to a small pilot trial from a Stanford research team. The study will be published June 1 in Biological Psychiatry.

From a press release I wrote to explain the new findings:

The antioxidant, called N-Acetylcysteine, or NAC, lowered irritability in children with autism as well as reducing the children's 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, MD, the primary author on the new study. "It can affect learning, vocational activities and the child's ability to participate in autism therapies."

NAC has some important potential advantages over other autism therapies. Right now, irritability in autism is often treated with second-generation antipsychotics. These drugs produce a greater decrease in irritable behaviors than the Stanford team saw with NAC, but also have severe side effects, potentially causing weight gain, involuntary motor movements and metabolic syndrome, which can lead to diabetes.

In addition, no medications exist to treat repetitive behaviors and other "core features" of autism such as social deficits and language impairment. If the findings about NAC's effect on repetitive behaviors are replicated in larger trials, NAC will be the first drug to treat one of the disorder's core features.

Hardan's team is now applying for funding for a large, multi-center trial of NAC. He told me:

One of the reasons I wanted to do this trial was that NAC is being used by community practitioners who focus on alternative, non-traditional therapies. But there is no strong scientific evidence to support these interventions. Somebody needs to look at them.

Previously: New imaging analysis reveals distinct features of the autistic brain

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