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Accelerating the discovery of new treatments for autism

Holland Bloorview Research Institute team members.

THE PROJECT

For this project, Evdokia Anagnostou and her team are trying out an innovative new approach which aims to accelerate the discovery of new treatments in ASD. They are testing novel compounds with already established early safety profiles simultaneously in children with autism and in multiple lines of mice bearing autism mutations. This represents the first co-clinical trial in humans and animal models in ASD. Using mice allows the team to isolate some of the individual causes of autism,and they can therefore test whether the drug works better in mice with one particular biology than mice with another. Extensive information on both human and mouse populations is collected for each trial to provide insight into the effectiveness of each compound being tested and to help in determining which subsets of patients would most benefit from each treatment. The team’s hope is that these co-clinical trials in children/youth and mice will lead to the creation of individual plans for each patient so that he or she can benefit from new treatments for ASD.

To date, the team has completed one trial in humans and its respective study in mice and analysis is currently underway. A new trial will be starting during the summer of 2018 in both children and mice and they have already completed another mouse study to complement a trial in children with ASD that was already completed before the grant was funded. The grant has also facilitated the creation of a community of basic scientists and clinical researchers that examine the issue from different perspectives to expedite discovery.

 

“Clinical trials are very difficult to fund and conduct. In addition, the cross species trials are the first of their kind in ASD and therefore a high-risk proposition that would not be considered by traditional funding sources. ”
– Evdokia Anagnostou