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Reaching for the Summit

Autism research is making great strides internationally in places such as Europe and Canada, due in large part to large, collaborative research systems established in those areas. These systems allow researchers across continents to share knowledge and work together toward mutually established research goals.  While there are some collaborative systems in the U.S., currently no large collaborative initiatives exist that integrate the top-down approach of clinical research and the bottom-up approach of basic research. How to establish such a system in the U.S. was the looming question that brought together dozens of leading American autism researchers at the third Thompson Center Research Summit in the fall of 2018.

Organized by Thompson Center researcher and University of Missouri professor Dr. David Beversdorf, these leading autism experts gathered to create a plan for modernizing how autism research is performed in the U.S.

“We as a nation have fallen behind other countries in this aspect of autism research production, due in large part to a lack of support for a large cohesive collaborative structure which allows researchers to better work together,” Beversdorf said. “We joined as a research summit to find a new way forward to gain ground in advancing autism research here in the U.S.”

However, once the summit began and the topic of creating a similar research collaborative structure as other countries was discussed, the summit took an unexpected turn.

“The consensus of the room was that while it is true that we as a nation were already behind in this collaborative area, the best approach may not be trying to recreate what is already being done.” Beversdorf said. “Instead, why not devote our time and resources in directions where we as a nation already have a foot forward and advance those areas?”

As a result of the summit, participants formed a few subgroups to advance several of these very areas. First, a group was organized to work on organizing existing and future autism biomarker databases so that they can be integrated.

“Thanks to monumental efforts of several research organizations, we have a growing list of databases across the country that hold valuable biomarker data which may help us understand the various causes of autism,” Beversdorf said. “If we can integrate these different data sets, it will allow for expanded research into these biomarkers, which will further help determine what factors lead to very specific subsets of autism. However there are significant barriers to accomplishing this, ranging from differences in formatting to differences in types of data registered, which need to be addressed.”

Another subgroup from the summit will work on expanding the capacities of systems like the Preclinical Autism Consortium for Therapeutics, where a range of rodent models targeting the various known causes of autism can be systematically compared for responses to treatment, perhaps even expanding models to include induced pluripotent stem cells, where a patient’s own cells can be utilized to examine the cellular responses to treatment.

Finally, a group led by Dr. Beversdorf will explore the possibility of writing a paper about the need for a new stage in clinical trials called an open label biomarker exploratory trial. This stage would identify autism research participants who have specific biomarkers that would most likely be affected by a specific clinical trial.

“Autism is a very diverse disorder, with many different subsets, most of which we are still working to identify,” Beversdorf said. “Often, clinical trials for autism therapies are unsuccessful overall, resulting in a negative study, but the drug did work for a specific subset of patients with autism.  We haven’t done a good job of identifying and targeting our participation pool to those subsets that may be most positively affected. So when a trial comes back as a failure, it may still have success for that small population, but it looks unsuccessful because the results for the rest of the population outweighed the benefits for this specific subset of autism.”

Despite taking an unexpected turn and heading in an unforeseen direction, the third Thompson Center Research Summit can be considered a success.

“It was heartening to hear from other leading autism research experts and to come to the consensus that we as a group can find novel ways of understanding the heterogeneity of autism, harnessing the outcomes of the European and Canadian collaboratives, as well as other sources,” Beversdorf said. “Rather, we were able to find ways to integrate our strengths with the previous efforts of others and continue moving forward in directions that will be most productive.”

The 2018 Thompson Center Research Summit took place as a part of the 13th annual Thompson Center Autism Conference in St. Louis. The summit was funded by the Thompson Center, the University of Missouri School of Medicine, BioNexus KC, and the University of Missouri Radiology department.