At autism summit, neuroscience meets pharmaceutical research to explore new treatments

By Adrienne Cornwall

COLUMBIA, Mo. (Oct. 22, 2014) – A group of 20 clinicians and researchers gathered at University of Missouri on Saturday for a series of working sessions and a special lecture focused on integrating biomarker and basic neuroscience research with pharmaceutical treatments for autism spectrum disorders (ASD).

“We don’t have any approved drugs at this time to treat the core features of autism,” said organizer Dr. David Beversdorf, associate professor at MU and research faculty at the Thompson Center for Autism and Neurodevelopmental Disorders.

ASD core features include difficulty with social interactions, communication challenges and repetitive behaviors. However, autism often occurs alongside other conditions such as anxiety, attention disorders, and developmental or learning disabilities as well as physical issues like sleep problems, gastrointestinal disturbances and immune system dysfunction.

Many physicians currently prescribe two common antipsychotics, risperidone and aripiprazole, to help treat common behavioral symptoms in autistic patients.

Some drugs that have been used off label have helped some patients but haven’t met criteria for success in drug testing. Because the combination of symptoms and their severity is different for every patient, the drugs studied thus far don’t work for all autism patients and have varying levels of side effects.

It is becoming increasingly clear that there are different forms of autism that likely have different causes, said featured speaker Dr. David Amaral, professor of psychiatry at University of California-Davis.

“The clinical trials for new drugs in autism have thus far failed in the United States,” Beversdorf said. “When you lump all the patients together, you don’t get a great response.”

By using biomarkers, such as the neuroimaging data being studied by Amaral, the principal investigator on the Autism Phenome Project (APP), researchers hope to better understand which groups of ASD patients will respond to particular drugs. It also refines how neuroscientists could further focus their tests with mouse models to uncover why and how those medications work in the neural pathways in those subgroups.

Amaral’s lecture on Friday about the APP highlighted the neuroimaging findings being collected by the study. By comparing these detailed pictures of the brains of typically developing children against children with autism and their range of symptoms, the project has found different patterns of altered brain development in different groups of young children with autism.

“By defining distinct types of autism, it will be faster and easier for scientists to study the causes of each type. In some cases, this may end up leading to preventions. We also believe that defining types of autism will lead to more effective treatment,” said Amaral, research director of the UC-Davis MIND Institute.

In addition to the lecture, the working group of 20 participants agreed to draft a position paper about how to integrate biomarker data into future clinical trials for medicines that can help treat the core symptoms of autism, and to integrate this with basic neuroscience research including animal models.

The summit group’s position paper, expected to be complete within a few months, will describe how researchers in the U.S. should approach integrating traditional neuroscience methods, advanced techniques like neuroimaging, and transformative research like genetics, epigenetics, and environmental epidemiology, to work together to help more patients with autism.

“The paper would provide a guiding vision for next-generation research on autism in the U.S.,” Amaral said.