To fight against dementia, the government takes a cannon … a pill



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"I'm worried since dinner," he says. "I'm afraid this is the beginning of an aggravation."

Like all dementia, including Alzheimer's disease, there is no cure. Treatments to slow the disease have limited effectiveness.

But a new clinical trial for a cheap drug that Hughes is involved in is "keeping a little hope alive," he said.

Scientists hope that the drug, sodium selenate, will slow the progression of the disease. Dr. Hughes is currently participating in a Phase 1 trial of the drug through Royal Melbourne Hospital.

On Friday, the federal government announced $ 1.6 million in funding from the Future Fund for Medical Research for a Phase Two clinical trial on selenate for frontotemporal dementia.

A phase two trial is one of the last steps before a drug is transferred from the lab to the lab – so the results are promising.

But it's a huge if. For several reasons.

First, billions of dollars have already been spent on unsuccessful clinical trials of anti-dementia drugs. The disease is extremely difficult to treat – much more difficult than cancer, it seems.

And the first try, the first step, is still going on. No results – positive or negative – have been published yet.

Finally, a small phase two trial of selenate in patients with Alzheimer's disease, published in 2016, showed that this did not slow cognitive degeneration.

Nevertheless, the team behind the new test is optimistic – the compound works well in animal studies and on the laboratory bench. And the Phase 1 trial at Alfred Hospital suggests a slowing down of brain degeneration, says Professor Terry O'Brien, director of the study, who heads the neuroscience department at Monash University.

"Anecdotally, yes. Patients progressed but more slowly than expected. But we have no evidence, because we have no patients who have not been treated to compare, "explains Professor O'Brien.

Professor O'Brien hopes that the drug will be more effective on frontotemporal dementia than on Alzheimer's disease.

In Alzheimer's, the damage to the brain is caused by toxic entanglements called h-tau, which twist in the brain and stifle nutrient stores, but also by amyloid plaques, which destroy neurons.

In about 50% of cases of frontotemporal dementia, the damage is caused exclusively by h-tau entanglements. In animals, selenate seems promising to dissolve these tangles and reduce brain tau levels, which means that it may work better for this disease.

And the compound seems safe, unlike other anti-tau drugs that have been shown to be neurotoxic.

"The concept is appealing," says Professor Christopher Rowe, director of the Australian Dementia Network. But until now, none of the studies has provided solid evidence of the compound's efficacy, he said.

"In reality, only an improvement in cognition or a slowing down of cognitive decline counts."

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None of this debate matters much to Mr. Hughes and his wife, Ann. They know that to find a new treatment, you have to chase moonshots.

"There is no treatment – so we'll stand out and try everything we can," Ann says.

Liam is the science journalist of The Age and Sydney Morning Herald

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