Changing mouse behavior with drugs, Israeli lab aims at possible autism therapy

Hebrew U researchers see reduction of symptoms in mice when specific gene mutation is targeted by chemicals, though treatment for humans still far off

Nathan Jeffay is The Times of Israel's health and science correspondent

Illustrative: Laboratory mice. (toeytoey2530/Istock via Getty Images)
Illustrative: Laboratory mice. (toeytoey2530/Istock via Getty Images)

Jerusalem scientists said they have reduced autism-like symptoms in mice by giving them human drugs and other chemicals, in research they hope will pave the way for autism therapeutics.

Hebrew University geneticists are studying POGZ, one of many human genes that, if mutated, is thought to cause autism. They have produced mice with this mutation, which they say mirror behavioral traits of autistic people who have the mutation.

“In the lab we are already treating mice with chemicals that change the brain activity to compensate for the mutation and restore normal activity,” Sagiv Shifman, associate professor in the Department of Genetics at the Hebrew University of Jerusalem, told The Times of Israel.

Various chemicals are being used to see how the brain reacts to a set of stimuli. They include drugs used by humans, which he declined to name, given in minute doses. Shifman said a reduction in autism-like symptoms has been observed in mice given the treatments.

The mice study has not been published or peer-reviewed and is expected to continue for years before researchers begin looking into possible applications for therapeutics that could be given to humans.

But Shifman said his study is an important step toward the possibility of treating people with autism spectrum disorder caused by POGZ mutations and possibly other gene mutations as well.

In November, the peer-reviewed journal Nature Communications published a study from Shifman, his collaborator Prof. Yosef Yarom, and a research team that appeared to pinpoint how the POGZ mutation affects mice’s brains.

Prof. Illana Gozes, a Tel Aviv University autism expert who is unconnected to Shifman’s research, said the published study “paves the path toward the development of targeted POGZ potential therapies.”

For years, the researchers have closely monitored mice with the mutation to see exactly how their brains differed from other mice.

Sagiv Shifman, associate professor in the Department of Genetics at the Hebrew University of Jerusalem (courtesy of Hebrew University)

As the mice displayed hyper-social conduct, learning disabilities, growth delay, and unusual physical appearance, the researchers observed changes in the brain, particularly in the cerebellum. This brain region is responsible for motor function and is thought to also contribute to the development of many social and cognitive functions.

“We saw molecular changes and we saw changes in the physiology of this brain region. This is very important because previous research shows this brain region is likely to be important for various genes that cause autism, not just one,” said Shifman.

By getting a detailed picture of the neurological processes taking place, Shifman’s team was able to start theorizing about what chemicals may counter the processes and design experiments to conduct on the mice.

If the cerebellum is shown to be a target for autism treatment, he said, this would be a particular cause for hope, as it changes more after birth than most other brain regions. This could provide a window in early childhood for therapeutics to shape its development.

“It’s a part of the brain that changes after birth which means it’s potentially more amenable for therapy, and it may be possible to treat autism in the early years. So this research could possibly be very important.”

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