A specific antioxidant supplement may be an effective therapy for some
features of autism, according to a pilot trial from the Stanford
University School of Medicine and Lucile Packard Children's Hospital
that involved 31 children with the disorder.
The antioxidant, called PharmaNAC, contains pharmaceutical-grade
N-acetylcysteine and is specially-packed to preserve its potency.
PharmaNAC lowered irritability in children with autism as well as
reduced the children's repetitive behaviors. The researchers emphasized
that the findings must be confirmed in a larger trial.
Irritability affects 60 to 70 percent of children with autism. "We're
not talking about mild things: This is throwing, kicking, and hitting;
the child needing to be restrained," said Antonio Hardan, MD, 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 appears in the June 1 issue of Biological Psychiatry. Hardan is an associate professor of psychiatry and behavioral sciences
at Stanford and director of the Autism and Developmental Disabilities
Clinic at Packard Children's.
Finding new medications to treat autism and its symptoms is a high
priority for researchers. Currently, irritability, mood swings and
aggression, all of which are considered associated features of autism,
are treated with second-generation antipsychotics. But these drugs
cause significant side effects, including weight gain, involuntary
motor movements and metabolic syndrome, which increases diabetes risk.
By contrast, side effects of PharmaNAC are generally occasional and
mild, with gastrointestinal problems such as constipation, nausea,
diarrhea and decreased appetite. Most people report no side effects. It
is easy for children to take because the PharmaNAC tablet is dropped
into a small glass of water to make a fizzy drink, so the child can
take it without having to swallow a pill or capsule.
The state of drug treatments for autism's core features, such as social
deficits, language impairment and repetitive behaviors, is also a major
problem. "Today, in 2012, we have no effective medication to treat
repetitive behavior such as hand flapping or any other core features of
autism," Hardan said. PharmaNAC could be the first medication available
to treat repetitive behavior in autism — if the findings hold up when
scrutinized further.
The study tested children with autism ages three to 12. They were
physically healthy and were not planning any changes in their
established autism treatments during the trial. In the double-blind
study design, children received PharmaNAC or a placebo of identical
appearance and taste for 12 weeks. The product used was the
effervescent, pharmaceutical-grade preparation donated by BioAdvantex
Pharma Inc., the manufacturer.
Subjects were evaluated before the trial began and every four weeks
during the study using several standardized surveys that measure
problem behaviors, social behaviors, autistic preoccupations and drug
side effects.
During the 12-week trial, PharmaNAC treatment decreased irritability
scores from 13.1 to 7.2 on the Aberrant Behavior Checklist, a widely
used clinical scale for assessing irritability. The change is not as
large as that seen in children taking antipsychotics. "But this is
still a potentially valuable tool to have before jumping on these big
guns," Hardan said.
In addition, according to two standardized measures of autism mannerisms
and stereotypic behavior, children taking PharmaNAC showed a decrease
in repetitive and stereotyped behaviors.