Better Animal Models Lead to Better Treatments

Tuesday, August 7, 2012 View Comments

Geneticist Richard Paylor explains the importance of the animal models being developed with Autism Speaks support. 

Posted by Richard Paylor, PhD, professor of molecular and human genetics at Baylor College of Medicine, whose research is supported by grants from Autism Speaks

This week Autism Speaks announced that it initiated and is funding the development of new genetically modified rat models of autism spectrum disorder (ASD). My team at Baylor College of Medicine will help characterize these models in close partnership with the science staff at Autism Speaks and SAGE Labs. These models will provide autism researchers with important new tools for understanding the underlying biology of ASD and testing experimental medicines. Indeed, they will be among the first rat models made widely available to the field of autism research.

Why will rat models help advance autism research? In general, animal models are an important first step in testing whether promising new medicines might be safe and effective in people. For good reason, the U.S. Food and Drug Administration requires new experimental medicines be tested in animals before they advance to clinical trials. Because the biology of rats is so well characterized, they tend to be the preferred animal models for testing drug safety.

In addition, rats have several advantages for testing the potential effectiveness of drugs that might relieve autism’s disabling symptoms. Currently, autism research relies primarily on mouse models. But mice do not display the rich array of social behaviors we see in rats. Rats play, cuddle and groom each other. Like mice, rats communicate using ultrasonic noises that we can record in the lab. As a result, they make better models of “autism-like” animal traits. These include compulsive self-grooming and decreased social play and vocalization. This allows researchers to better measure how well a drug might relieve similar symptoms of repetitive behavior and reduced communication and sociability in people.

Of course, autism is a uniquely human condition, and animal models will never be a perfect tool for understanding human development and behavior. However, they can be an important place to start. Already the insights we’ve gained from studying autism-like traits in animals have greatly increased our scientific understanding. Improving the availability of better animal models will help the field increase the development of medicines that can relieve disabling symptoms.

Five years ago, my lab received a grant from Autism Speaks to collaborate with Seaside Therapeutics to test new drug compounds in a fragile X mouse model. Like many people living with fragile X, these mice display autism-like traits. Our research showed that, in these mice, several of the experimental compounds improved social interactions and reduced compulsive and anxiety-related behaviors. These compounds are now being tested in human clinical trials, with the hope that they will likewise prove safe and effective in easing disabling core symptoms of autism.

We believe that the new rat models will speed and expand our ability to develop safe and effective therapies for those struggling with autism spectrum disorder. As an autism researcher, I want to personally thank Autism Speaks and its donors for their continuing support.

Explore more of the studies we’re funding with our grant search, and read more news and perspective on our science page.