Autism is currently diagnosed using behavioral tests, which are designed to measure the social and communication difficulties and repetitive behaviors that define the disorder. Clinical trials testing new therapies for autism measure effectiveness using these same behavioral evaluations. But knowing which specific behavioral tests to use and how soon after treatment to apply them is a major challenge.

What’s more, behavioral tests designed to diagnose the disorder may not be sensitive enough to measure the effects of treatment that occur within the time frame of a clinical trial. Seaside Therapeutics, a Massachusetts-based company where I am vice president of research, is developing a therapeutic called arbaclofen to treat the core symptoms of autism.

On 3 December, we announced a partnership with Delivering Scientific Innovation for Autism (DELSIA), a funding venture launched by the advocacy and research organization Autism Speaks, to tackle this challenge.

Our goal is to find additional tests, such as biological markers in blood, that will better reflect how well a treatment is working, and that can easily be monitored multiple times over the course of a clinical trial. If we are successful, the results of our studies will provide two types of biomarkers, those that would help predict whether someone will respond to arbaclofen, allowing us to focus the clinical trials on this group, and those that would be a short-term measure of whether an individual is responding to arbaclofen.

We’ve already started the search by collecting blood samples from participants in our studies when they enrolled and again upon completion of the trial. We plan to analyze the exact DNA and RNA sequence for each participant, as well as a profile of the proteins in blood before and after treatment. 

It took more than two years to collect and bank the participants’ samples. Now, because of the collaboration between Seaside and DELSIA, the exciting work of generating and analyzing all these data can begin.

One unique aspect of this project will be the ability to integrate the DNA, RNA and protein data and generate a detailed and informed profile for each participant who responded to our treatment. Because arbaclofen is the most advanced treatment for autism in the clinical pipeline, this is an important first step in the application of personalized medicine for individuals with autism.

Aileen Healy is vice president of research at Seaside Therapeutics.