ASD is a group of neurodevelopment disorders characterized by three core behavioral features: stereotypical repetitive behaviors, impaired social interaction and deficits in communication. ASD is also characterized by a significant heterogeneity in genetic background, phenotype and clinical presentation.
It is a highly common condition with an estimated prevalence of 1 in 59 school-aged children in the United States (Centers for Disease Control and Prevention). Estimates for EU are 1 in 100. This equates to approximately 10 million patients in Europe and North America.
ASD has a significant impact on the long-term quality of life of people living with the condition and their families and represents a considerable burden to both healthcare economies and society. There is currently no cure for ASD.
Diagnosis is based on clinical behavioral observations, which does not give insight into the genetic or molecular basis of the disease. 80% of ASD cases are idiopathic (autism with no currently identifiable genetic cause). There are currently no laboratory diagnostic tests.
Only two drugs are currently approved for use in patients with ASD. These drugs only treat irritability that can be associated with the condition and not the core symptoms, and they have a number of side effects. In current clinical trials for idiopathic autism, the same drug is given to all patients who behave in a similar way. This ‘one size fits all’ approach has been a major contributor to the failure in trials conducted in the last decade.