Developing new pharmacotherapies for autism
Article first published online: 10 AUG 2013
© 2013 The Association for the Publication of the Journal of Internal Medicine
Journal of Internal Medicine
Volume 274, Issue 4, pages 308–320, October 2013
How to Cite
Institute of Psychiatry, London, UK; F. Hoffmann-La Roche Ltd, Basel, SwitzerlandDeveloping new pharmacotherapies for autism. J Intern Med 2013; 274: 308–320., , .
- Issue published online: 10 SEP 2013
- Article first published online: 10 AUG 2013
- Accepted manuscript online: 19 JUL 2013 04:02AM EST
- Medical Research Council, UK. Grant Number: G0400061
- EU Seventh Framework Programme. Grant Number: FP7/2007-2013
- drug development;
- personalized medicine;
- translational research
Developing new pharmacotherapies for autism spectrum disorder (ASD) is a challenge. ASD has a complex genetic architecture, several neurobiological phenotypes and multiple symptom domains. However, new opportunities are emerging that could lead to the development of ‘targeted’ and individualized pharmacological interventions. Here, first we review these important new insights into the aetiology and neurobiology of ASD with particular focus on (i) genetic variants mediating synaptic structure and functioning and (ii) differences in brain anatomy, chemistry and connectivity in this condition. The characterization of the genotypic and phenotypic differences underlying ASD might in the future be invaluable for stratifying the large range of different individuals on the autism spectrum into genetically and/or biologically homogeneous subgroups that might respond to similar targeted interventions. Secondly, we propose a strategic framework for the development of targeted pharmacotherapies for ASD, which comprises several different stages in which research findings are translated into clinical applications. The establishment of animal models and cellular assays is important for developing and testing new pharmacological targets before initiating large-scale clinical trials. Finally, we present the European Autism Interventions – A Multicentre Study for Developing New Medications (EU-AIMS) Initiative, which was set up in the context of the EU Innovative Medicines Initiative as the first European platform for integrated translational research in ASD. The EU-AIMS Initiative consists of academic and industrial partners working in collaboration to deliver a more ‘personalized’ approach to diagnosing and treating ASD in the future.