Monday, June 9, 2014

Mice are key players in study of autism drugs

By Louise Kinross

Holland Bloorview and SickKids have received $2.5 million from Brain Canada to conduct a five-year trial of new autism medicines in children and mice.

Testing drugs at the same time in humans and mice has produced promising results in cancer treatments and is a first for autism research.

Identifying the genetic change that causes a specific type of autism can predict whether it will respond to treatment. But “there are too many different types of autism that we can’t figure out the biological subtype based on looking at a child’s behaviour,” says lead investigator Dr. Evdokia Anagnostou, child neurologist at Holland Bloorview’s Autism Research Centre.

“We’re learning a lot about genes that map on different pathways, but in humans these genetic differences account for a very small number of kids,” Evdokia says. “So we don’t have enough kids for each of the genetic variations to study them properly.”

Enter the mice. “We can engineer lines of mice that carry a specific genetic difference associated with autism and then study their brains and responses to treatment.”

Over the five-year period, two drugs will be trialled in children and six in mice. After two-and-a-half years “the most promising compounds in the mice will be put in the humans and we will continue testing other compounds,” Evdokia says.

Being able to test drugs in mice with a specific genetic mutation “means we can take drugs that exist for a completely different reason, that have nothing to do with autism or development, but which we know affect the pathway we’re looking at.”

Human studies will be done at Holland Bloorview, McMaster Children’s Hospital and the Lawson Research Health Institute. The mice trials will be run at SickKids.


While I understand that this is advancement in science, I am disturbed that this money is being spent on potentially drugging our children rather than providing us with supports that we need for helping them 'recover', such as providing coaching for parents on creating opportunities for interacting with our children. This has been shown in scientific research, such as the study at York University (MEHRI group) to physically change the brains of autistic children to resemble more "typical" developing brains and thus show less signs of autistic behaviours. Autism literally goes away for many kids as the brain pathways are enticed to develop more "typically" and this is where support for parents is most needed, imho... Prevention rather than treatment, although both have their value.

I always find that the position taken by the parent is fundamentally based on their own experience with their own child's form of ASD. There are many people with ASD that have a far more severe disability and for whom this type of study is important.

As you yourself note, a study at York has already looked at the approach you personally prefer. So therefore funds have already gone towards such a study at York, and some students can benefit from such an approach - but not ALL people with ASD will benefit from it. This is reason alone for recognizing that many different approaches are needed.

Moreover, there are interesting parallels between ASD brain and Alzheimer brain - such as build up of certain proteins in the brain. Given that our society is also facing a crisis in coming years with an aging population including those suffering dementia (75% dementia patients are women). To do research that could potentially benefit both ASD and Alzheimers is something worthwhile.