How Can Mice Help Advance Autism Research?

January 1, 1970

A first-of-its-kind co-clinical trial in humans and animal models with autism, leading to precision medicine, by Dr. Evdokia Anagnostou. Un essai co-clinique inédit sur des humains et des modèles animaux atteints d'autisme, menant à une médecine de précision, par le Dr Evdokia Anagnostou.

Thanks to Brain Canada, scientist Dr. Evdokia Anagnostou (right) does novel research that may lead to more targeted therapy for children with autism spectrum disorder.

Some children with autism respond to certain treatments and some don’t. Finding the right therapy depends on the particular biology of each individual, and this is difficult to determine. There are more than 1,000 genes that may be responsible for autism.

A few years ago, two early- career scientists – Dr. Evdokia Anagnostou, who works with children who have autism, and Dr. Jason Lerch, who works with laboratory mice – decided they could find more targeted and effective ways to treat autism by working in tandem. The concept of a co-clinical trial with children and mice was pretty radical. “The idea was ‘out there’,” said Dr. Anagnostou. “It was so novel that we knew it may not work, but thought that if it did work it would have a huge impact in the field.”

Brain Canada embraced the idea and helped to fund the research. Now, the duo has finished their first co-clinical trial, with promising results, and is working on their second.

It was so novel that we knew it may not work, but thought that if it did work it would have a huge impact in the field.

Dr. Anagnostou and Dr. Lerch assess children using brain imaging and genetic testing to determine their particular biological causes of autism. At the same time, they test various therapies in different mouse populations with a range of biological causes of autism. When certain mice respond well to therapy, the idea is to then match those treatments to those biologies in children.

“This was a first-of-its-kind co-clinical trial in humans and animal models with autism,” says Dr. Anagnostou, “It is a step towards precision medicine.” She is enthusiastic and hopeful that this bold project will help advance autism research and lead to better more precise treatments for individual patients.