Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental condition characterized by core symptoms of challenges in social interactions and repetitive and restricted behaviors and interests, in addition to common comorbidities (e.g., epilepsy and depression). The combination of core symptoms and comorbidities significantly reduces the quality of life and the life span of individuals with ASD. Currently, there are no effective drug treatments for the core symptoms. Key factors that have hampered progress in clinical research include: 1) limited understanding of the underlying pathophysiology(ies); 2) lack of successful translation from animal models to humans; 3) testing of drugs with specific actions in biologically heterogeneous populations; 4) in Europe, limited expertise of many ASD centers in running large-scale clinical trials; and 5) trial designs (e.g., placebo effects).

The Autism Innovative Medicine Studies-2-Trials (AIMS-2-Trials) is a large-scale collaborative research project that involves 48 partners across 14 countries from academia, industry and the nonprofit sector. It is led by a partnership between academia and industry, represented by project leads Declan Murphy and Will Spooren, respectively.

AIMS-2-Trials will build on the results of the European Autism Interventions – A Multicentre Study for Developing New Medications (EU-AIMS), a five-year grant that was previously awarded by the Innovative Medicines Initiative to investigate autism mechanisms and opportunities for translational research.

The goals of the project include: 1) aligning global resources to validate and qualify stratification biomarkers from infancy to adulthood; 2) developing objective outcome measures that can be used in trials; 3) creating a European-wide clinical-trials network that reliably carries out studies able to support filings to the European Medicines Agency and the U.S. Food and Drug Administration; 4) carrying out better-targeted clinical trials linked to other international efforts, including quick wins or ‘fast fails’ of ineffective agents; and 5) translating molecular mechanisms and drug effects between preclinical models and particular subtypes of ASD.

AIMS-2-Trials has received funding support and in-kind contributions from the Innovative Medicines Initiative, the European Union’s Horizon 2020 research and innovation programme, SFARI, Autism Speaks, Autistica and the European Federation of Pharmaceutical Industries and Associates (EFPIA).