Autism Spectrum Disorders (ASD): European Situation
Autism Spectrum Disorders (ASD): The European Situation 2016
- No major strategy defined within Europe
- No major or concerted efforts in drug discovery
- No pre-clinical network
- No clinical trial network
- No translational network
- No regulatory strategy
- Late diagnosis and poor awareness (including in adults)
- Poor knowledge of patients needs across life-course (teens into adulthood)
- Wide range in treatment strategy with no evidence of efficacy
Challenges for the basic and clinical translational science field in Europe
There are no effective pharmacological treatments for the core symptoms of autism spectrum disorder (ASD), and our understanding of the pathophysiology of the disease is poor. Research is hampered by a lack of valid and reliable cellular assays and animal models; an absence of tests that demonstrate efficacy in healthy volunteers from childhood to adulthood; and the reliance of clinical trials on biologically heterogeneous groups of patients as operationally-defined by DSM/ICD10 categories. Further, even if novel treatments were developed, there is no EU platform to test them clinically. Despite these limitations, the recent identification of genetic risk factors for ASD provides unique opportunities to substantially improve this situation. We therefore propose an integrated, translational, effort to achieve key objectives for ASD research, which will deliver new research tools and standards for clinical development, and pave the way for drug discovery and clinical trials. .
We need to harness these new developments to develop treatments that are driven by the likely biological basis of ASD, and tests and tools that identify and stratify patients with distinct disease subtypes.
A concerted effort of key stakeholders is needed: Private Public Partnership.
Basic and clinical translational science in Autism Spectrum Disorders (ASD): What EU-AIMS reached so far…
- Gaining scientific qualification advice from the European Medicines Agency (EMA) for the LEAP biomarker protocol
- Developing a clinical network of 93 clinical and/or research ASD centres spread across 37 countries
- Surveying the use of diagnostic assessments instruments in clinical sites across Europe