Reducing tau protein in brain ‘can help combat epilepsy’

Posted Aug 14 2014 in Brain science; genetics

A new study has suggested that reducing the level of a certain protein in the brain can help to combat Dravet syndrome, a severe form of childhood epilepsy.

Researchers from the Gladstone Institute of Neurological Disease in the US conducted a study in which levels of the protein tau were reduced in the brains of genetically-engineered lab mice with Dravet syndrome by knocking out the gene associated with its production.

According to results published in the Annals of Neurology, the deletion of one copy of the gene resulted in substantial improvements in most symptoms, with the removal of both copies eliminating them almost completely.

Among the beneficial effects were significant reductions in both spontaneous and heat-induced seizures. Network activity in the brain also returned to normal levels, while learning and memory deficits and behavioral abnormalities in the epilepsy-affected mice were also lessened.

Previous research has shown that lowering tau levels can reduce abnormal brain activity associated with Alzheimer’s disease, but this is the first study to demonstrate that the same is true of intractable genetic epilepsy.

The team now wishes to develop tau-lowering therapies for use in human patients that can be evaluated in preclinical trials.

Senior author Dr Lennart Mucke, director of the Gladstone Institute of Neurological Disease and a professor of neurology and neuroscience at the University of California, San Francisco, said: “We suspected that this approach might be beneficial in Dravet, but we couldn’t be sure because of the severity of this syndrome and the corresponding model. We are thrilled that our strategy was so effective, but a lot more work is needed to advance it into the clinic.”

Dravet syndrome results from a specific genetic mutation affecting sodium channels in the brain and is recognised as one of the hardest-to-treat forms of childhood epilepsy, causing frequent seizures, cognitive impairments, behavioural problems and a high risk of sudden death.

Though it is rare, affecting only one or two children with epilepsy out of every 500, the ineffectiveness of current therapy options for this condition make it important that new treatment approaches are created.

Posted by Anne Brown

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