News Feature | April 3, 2014

Experimental AntiCancer Reverses Schizophrenia In Teenage Mice

By Estel Grace Masangkay

Researchers from John Hopkins Medicine reported that an experimental cancer drug induced reversal of schizophrenia-related behaviors in adolescent mice afflicted with the rodent version of schizophrenia.

The anticancer compound, called FRAX486, belongs to the class of compounds known as PAK inhibitors which have been shown to offer some level of protection from brain damage due to Fragile X syndrome in animal experiments. Fragile X syndrome is an inherited disease marked by mental retardation in humans. PAK inhibitors have been considered as potential treatments for Alzheimer’s disease and even cancer.

In the study conducted by researchers from John Hopkins, the compound seemed to halt an uncontrollable biological pruning process in the schizophrenic brain during which healthy and crucial neural connections are unnecessarily attacked and destroyed. The researchers were able to partially restore disabled neurons using the investigational anticancer compound in adolescent mice.

Akira Sawa, professor of psychiatry and behavioral sciences at the Johns Hopkins University School of Medicine and leader of the study, said, “By using this compound to block excess pruning in adolescent mice, we also normalized the behavior deficit. That we could intervene in adolescence and still make a difference in restoring brain function in these mice is intriguing.”

The findings in adolescent mice were especially significant as schizophrenia usually manifests itself in late adolescence and early adulthood in humans. By using the compound to reduce PAK activity, the researchers were able to protect against excess biological pruning and helped allow restoration of affected areas. The test mice were examined on the 35th and 60th day of their life cycle which is the equivalent of adolescence and young adulthood. The subjects showed improvement after being treated with small doses of the PAK inhibitor.

“Drugs aimed at treating a disease should be able to reverse an already existing defect as well as block future damage. This compound has the potential to do both,” Professor Sawa said.

A report on the researchers’ work was published online in the Proceedings of the National Academy of Sciences last March.