Rationale and Objective: Most efforts at treatment of autism with drugs are directed at specific symptoms such as agitation, anxiety, obsessive behaviors, and depression. We do not yet have any drug proven to affect the core components of autism, such as language and social interaction. Previous large drug trials hoping to impact these core components of autism have failed. However, our work has explored the effects of the drug propranolol, widely used to control rapid heart rates, treat migraines, and help with tremor and performance anxiety. We have shown that propranolol helps language and social interaction after single doses are given. Now we wish to move forward to see if there is a benefit with regularly taking propranolol, as would be done in clinical practice.

Types of Patients Helped, and How It Will Help: We will explore the effects in two groups of patients. First, as our initial studies examined adolescents and young adults with single doses, we will see if these same benefits occur with regular doses in this age group. Second, the impact of a drug improving language and social interaction may be greater if it occurs earlier in life, while the child is early in the process of learning. Therefore, we will also see whether propranolol can have the same benefit in younger children.

Application, Benefits, and Risks: We would primarily expect a beneficial effect on social interaction, but we will also examine effects on language and overall functioning. We will also determine whether stress reactivity measures and brain imaging markers of autism can predict who will respond best, for the goal of ultimately developing individualized treatment, leading to the ability to know who is most likely to respond to propranolol. The potential risks with propranolol include trouble breathing in individuals with asthma, slowing of the heart rate, tiredness, stomach irritation, sadness, and troubles with sexual performance. Individuals with active problems with asthma, depression, and low heart rate or blood pressure will not be allowed to participate, and the ones that do participate will be informed of these risks.

Impact: The goal of this line of work is to impact autism spectrum disorder (ASD) patient care by resulting in the development of a new treatment option for core components of ASD, which does not currently exist. It may also result in markers to predict who is most likely to respond. Finally, as this agent is widely available in a generic form, it will increase access of care to the underserved.