TORONTO, June 1, 2012 /CNW/ - 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
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
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.
"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," Hardan said. "But there is no strong scientific evidence to
support these interventions. Somebody needs to look at them."
Hardan cautioned that the NAC for sale as a dietary supplement at
drugstores and grocery stores differs in some important respects from
the individually packaged doses of pharmaceutical-grade NAC used in the
study, and that the over-the-counter version may not produce the same
results as PharmaNAC. "When you open the bottle from the drugstore and
expose the pills to air and sunlight, it gets oxidized and becomes less
effective," he said.
Although the study did not test how PharmaNAC works, the researchers
speculated on two possible mechanisms of action. PharmaNAC increases
the capacity of the body's main antioxidant network, which some
previous studies have suggested is deficient in autism. In addition,
other research has suggested that autism is related to an imbalance in
excitatory and inhibitory neurotransmitters in the brain. PharmaNAC
could modulate the glutamatergic family of excitatory
neurotransmitters, which might be helpful in autism.
The scientists are now applying for funding to conduct a large,
multicenter trial in which they hope to replicate their findings.
"This was a pilot study," Hardan said. "Final conclusions cannot be made
before we do a larger trial."
"BioAdvantex is delighted with the promise that this research shows for
PharmaNAC in treating irritability and repetitive behaviours in
children with autism," said David Aiello, President, BioAdvantex
Pharma. "We are committed to continuing to support research of this
condition which affects many Canadians, both adults and children."
Hardan's collaborators at Stanford were Lawrence Fung, MD, a psychiatry
resident; Robin Libove and Surekha Nair, MD, social science research
assistants; postdoctoral scholar Tetyana Obukhanych, PhD; Lenore
Herzenberg, DSc, professor of genetics and member of the Stanford
Cancer Institute; and Rabindra Tirouvanziam, PhD, a former instructor
in pediatric pulmonary medicine at Stanford who is now at the Emory
University School of Medicine.
Information about the Stanford Autism Center at Packard Children's
Hospital and the Department of Psychiatry and Behavioral Sciences,
which also supported the research, is available online at http://autism.lpch.org and http://psychiatry.stanford.edu.
Thiolex Development Corporation has exclusively licensed the technology
behind the use of N-acetylcysteine in autism to BioAdvantex Pharma
Inc., a related company.
For further information:
David Aiello, President
BioAdvantex Pharma Inc.