| Reference | 1. Drug Healthc Patient Saf. 2014 May 10;6:69-76. doi: 10.2147/DHPS.S39595.
eCollection 2014.
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Clinical potential, safety, and tolerability of arbaclofen in the treatment of
autism spectrum disorder.
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Frye RE(1).
<br>
Author information: <br>
(1)Arkansas Children/’s Hospital Research Institute, Department of Pediatrics,
University of Arkansas for Medical Sciences, Little Rock, AR, USA.
<br>
Autism spectrum disorder (ASD) is a behaviorally defined disorder which has
increased in prevalence over the last two decades. Despite decades of research,
no effective treatment is currently available. Animal models, as well as other
lines of evidence, point to abnormalities in the balance of cortical excitation
to inhibition in individuals with ASD, with this imbalance resulting in an
overall increase in cortical excitation. To reduce cortical excitatory glutamate
pathways, arbaclofen, a selective agonist of the gamma aminobutyric acid receptor
type B, has been developed. This article reviews the evidence for this treatment
for ASD using a systematic review methodology. Overall, a systematic search of
the literature revealed 148 relevant references with the majority of these being
review papers or news items that mentioned the potential promise of arbaclofen.
Five original studies were identified, four of which used STX209, a form of
arbaclofen developed by Seaside Therapeutics, Inc., and one which used
R-baclofen. In an animal model, treatment of Fragile X, a genetic disease with
ASD features, demonstrated a reversal of behavioral, neurological, and
neuropathological features associated with the disease. One double-blind,
placebo-controlled study treated children and adults with Fragile X. Results from
this study were promising, with signs of improvement in social function,
especially in the most severely socially impaired. Two studies, one open-label
and one double-blind, placebo-controlled, were conducted in children,
adolescents, and young adults with ASD. These studies suggested some improvements
in socialization, although the effects were limited and may have been driven by
individuals with ASD that were higher-functioning. These studies and others that
have used arbaclofen for the treatment of gastroesophageal reflux suggest that
arbaclofen is safe and well-tolerated. Clearly, further clinical studies are
needed in order to refine the symptoms and characteristics of children with ASD
that are best treated with arbaclofen.
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2. ACS Chem Neurosci. 2011 Aug 17;2(8):381. doi: 10.1021/cn200061g. Epub 2011 Jul 6.
<br><br>
ACS chemical neuroscience molecule spotlight on STX209 (arbaclofen).
<br><br>
Hopkins CR(1).
<br>
Author information: <br>
(1)Department of Pharmacology and Vanderbilt Center for Neuroscience Drug
Discovery, Vanderbilt University Medical Center, Vanderbilt University,
Nashville, Tennessee 37232-6600, USA.
<br>
STX209 (arbaclofen) is a γ-amino butyric acid type B (GABA(B)) receptor agonist
from Seaside Therapeutics currently in clinical trials for autism spectrum
disorders (ASD). The company has initiated a phase 2b study after positive
results from a phase 2a trial, announced September 2010
(http://www.seasidetherapeutics.com/sites/default/files/STX209_ASD_P2b
Trial_Initiation%206%2021%202011%20Final.pdf).
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