Objective
Despite dramatic advances in molecular and imaging technologies, there are currently no effective pharmacological treatments for the core symptoms of autism spectrum disorder (ASD). Major obstructions to this include a lack of aetiologically-driven or pathophysiologically-accurate animal models; an absence of tests that indicate efficacy; and reliance of clinical trials on DSM/ICD10 categories which provide a collection of biologically heterogeneous patients. Further, even if novel treatments are developed there is no EU platform to clinically test them. Our hypothesis is that a focus on cross-species endophenotypes, finding biologically-homogenous groups of patients, and developing a clinical research network will overcome the limitations in target identification, early triage and clinical trials. Hence, leading European institutions and three SMEs will partner with the EFPIA to: a) develop in vitro models, and animal models that carry confirmed genetic risks, and in these animals to focus on cross-species endophenotypes (e.g. cognitive function, electrophysiology) to facilitate new drug discovery; b) validate the use of sMRI and fMRI-based endophenotypes in genetically-selected healthy volunteers, infants at risk for ASD, and children and adults (including twins) with and without ASD, as early and surrogate markers for efficacy; and to combine this with PET approaches to provide guidance regarding optimal clinical trial design; and c) identify biomarkers that can be used to stratify patients within an umbrella DSM-diagnosis, thus allowing for targeted clinical trials, individualized treatment and back-translation of subgroup-specific biomarkers into preclinical drug discovery. To increase the chance of a breakthrough we will implement new analytical approaches (e.g. support vector machine learning algorithms) and will actively collaborate with patient groups and other international efforts (e.g. the Autism Genetic Resource Exchange (AGRE)).
Fields of science
- medical and health sciencesbasic medicinepharmacology and pharmacydrug discovery
- natural sciencesbiological sciencesneurobiologycognitive neuroscience
- medical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugs
- natural sciencescomputer and information sciencesartificial intelligencemachine learning
Call for proposal
IMI-JU-03-2010
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Coordinator
4070 Basel
Switzerland
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Participants (26)
WC2R 2LS London
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68159 Mannheim
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6525 XZ Nijmegen
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CB2 1TN Cambridge
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101 Reykjavik
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3584 CX Utrecht
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4051 Basel
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75724 Paris
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80333 Muenchen
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80539 Munchen
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69117 Heidelberg
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2750 Ballerup
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17177 Stockholm
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RG21 4FA Basingstoke
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2340 Beerse
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91190 GIF-SUR-YVETTE
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1752 Villars-sur-Glâne
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WC1E 7HX London
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00128 Roma
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08540 Princeton Nj
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CT13 9NJ Sandwich
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75015 PARIS 15
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89081 Ulm
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6709 PA Wageningen
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101 Reykjavik
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75008 Paris
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