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Compounds and methods for treating autism spectrum disorders

a technology for autism spectrum disorders and compounds, applied in the field of compounds and methods for treating autism spectrum disorders, can solve problems such as inability to meet patients, and achieve the effect of enhancing the sociability of mi

Inactive Publication Date: 2010-06-10
BIOCODEX INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

[0009]The present invention arises from the unexpected observation that resolution of autism symptoms occurs in patients after initiation of treatment with stiripentol. This observation can be confirmed by studies in animal models that mimic characteristic features of autism such as the models described by Moy S S et al, Behav. Brain Res. 176:4, 2007 and McFarlane H G et al, Genes, Brain and Behavior, 1601:1, 2007. Thus, in a mouse model of autism (BALB / c mice), the inventors have shown that stiripentol enhanced the sociability of the mice thereby indicating that stiripentol would be useful in the management of autism.
[0012]Without willing to be bound to any particular theory, it is believed that stiripentol can alleviate the consequences of the mutations of the GABA receptor subunits associated to autism.

Problems solved by technology

The concept is that autism results from an imbalance between inhibitory GABAergic and excitatory glutamatergic pathways.

Method used

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  • Compounds and methods for treating autism spectrum disorders
  • Compounds and methods for treating autism spectrum disorders
  • Compounds and methods for treating autism spectrum disorders

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[0039]The low level of sociability in mice of the BALB / cByJ (hereafter BALB / c) inbred strain is considered to be relevant to autism (Brodkin (2007) Behav Brain Res 176:53-65; Moy et al. (2008) Behav Brain Res 188:178-194; Brodkin et al. (2004) Brain Res 1002:151-157). Furthermore, BALB / c mice show other phenotypes with possible relevance to autism, including relatively high levels of anxiety and aggressive behaviors, large brain size, underdevelopment of the corpus callosum, and low levels of brain serotonin synthesis (Brodkin (2007) Behav Brain Res 176:53-65; Moy et al. (2007) Behav Brain Res 176:4-20; Sankoorikal et al. (2006) Biol Psychiatry 59:415-423; and Moy et al. (2008) Behav Brain Res 188:178-194).

[0040]The purpose of this study was to examine the effects of stiripentol on the social behavioural in BALB / c mice compared to C57BL / 6J (C57) inbred mice. The social investigatory behaviour of mice was measured by the amount of time that the “test” mouse (BALB / c and C57 mice) enga...

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Abstract

The present invention relates to a compound of the following formula (I):or a pharmaceutically acceptable salt thereof,for use in the prevention or treatment of autism spectrum disorders (ASD).

Description

[0001]The present application claims the benefit of U.S. provisional application 61 / 120,935 filed on Dec. 9, 2008, which is incorporated herein by reference.FIELD OF THE INVENTION[0002]The present invention relates to compounds and methods useful for preventing and treating autism spectrum disorders (ASD).BACKGROUND OF THE INVENTION[0003]Autism is a cognitive disorder associated with three diagnostic characteristics: abnormal social interaction, impaired communication and repetitive patterns of behavior (Geshwind D H and Levitt P, Curr. Opin. Neurobiol. 17:103, 2007). Autism is part of a spectrum of disorders called autism spectrum disorders (ASD). Autism has an incidence of approximately 1 in 1,000 individuals while ASD has a higher incidence of 1 to 6 per 1000 individuals. Onset is typically prior to three years of age.[0004]There is a consensus that autism is a neurobiological disorder (Luke Y and Tsai M D, Psychosomatic Medicine 61:651, 1999; Levy & Schultz, Lancet 374:1627, 200...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): A61K31/36
CPCC07D317/54A61K31/36A61P25/00
Inventor HUBLOT, BERNARDLEVY, RENELE GUERN, MARIE-EMMANUELLE
Owner BIOCODEX INC
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