Aussie researchers at the forefront of massive dementia breakthroughs
Dementia is one of the very worst diagnoses a human can face – but Aussie scientists are on the cusp of a game-changing breakthrough.
A dementia diagnosis has, for many, long been among the very worst of news to hear from a doctor.
More than 50 per cent of Australians fear developing dementia, according to Body+Soul’s Health of the Nation report released earlier this year, which found that fear was greater among older Australians and women.
The number of people living with dementia worldwide is projected to reach around 153 million by 2050, while in Australia, it is estimated more than 433,000 people are currently living with dementia, with that number set to reach one million by 2065.
In a career marked by witnessing more than his fair share of heartbreaking outcomes, Neuroscience Research Australia (NeuRA) CEO and Institute Director Professor Matthew Kiernan said the field is on the brink of massive breakthroughs.
Drugs, early screening tests and … a vaccine?
“Right now we’re starting to see the emergence of a very focused therapy,” Prof Kiernan said. “The first of these, which was approved by the TGA here in Australia in May, is a monoclonal antibody directed against amyloid, the proteins that build up in the brain with Alzheimer’s disease.”
Trials of the drug showed it was able to slow decline in those with mild cognitive impairment by 35 per cent, with the best results coming if it was administered early in the condition’s progression.
The treatment, Donanemab [marketed in Australia as Kisunla], is approved for adults with early symptomatic Alzheimer’s disease who have the confirmed presence of amyloid plaque in the brain.
An application to have Kisunla added to the Pharmaceutical Benefits Scheme was knocked back by the Pharmaceutical Benefits Advisory Committee (PBAC) in August.
“While clinical trials demonstrated that Donanemab can potentially delay the progression of early Alzheimer’s disease by approximately six weeks, the PBAC noted there is a lack of consensus among clinicians that these results would translate into meaningful improvements for patients,’” the committee said.
“The PBAC considered the potential benefits too small and uncertain to justify the burden of this treatment on both patients and the health system”.
Patients prescribed the dug will need to pay privately, with treatment costs ranging between $40,000 and $80,000 a year.
Despite the setback, Prof Kiernan – who is throwing his support behind news.com.au and The Australian’s Think Again campaign – said a groundbreaking clinical trial aims to prevent the onset of Alzheimer’s disease altogether in people with high genetic risk factors.
“It’s very exciting and could be a game-changer,” he said.
“The study is called DIAN-TU (which stands for ‘Dominantly Inherited Alzheimer’s Network Trials Unit).
“Neurologist Dr Emma Devenney is running that trial, and we’re treating people with genetic abnormalities linked to Alzheimer’s disease.
“They’ve started on Donanemab now, and we’re trying to see if we can prevent the onset in those individuals,” Prof Kiernan said.
It’s helpful, Prof Kiernan said, to think of the monoclonal antibody treatment as working in a similar way as vaccines.
“In fact, the cutting edge science at the moment is to look into vaccination.
“So could we vaccinate children at the age of one, two or three, and never have Alzheimer’s or dementia in the future? That’s where the field is, right at the moment,” he said.
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“GSK [pharmaceutical company] here in Australia – they’ve got a huge group of scientists globally – and that’s their focus.
“They want to come up with the vaccine for Alzheimer’s disease. So it is conceivable that this won’t be a condition in the future.
“I’m talking probably many decades before that could happen and it’s still hypothetical, so it’s still a bit more science fiction, but it’s worth understanding that industry and pharma are investing a lot of money in that today.”
A blood test for dementia?
Of course, Donanemab is specific to only one type of dementia: Alzheimer’s.
“There’s also dementia associated with Parkinson’s disease, for example that’s due to a different protein, alpha synuclein. Then there’s a young onset Frontotemporal dementia, and that’s a different protein again, and then there is also vascular dementia, and that’s similar to vascular disease in your heart, so blood pressure, cholesterol, diabetes, smoking – all of those risk factors,” Prof Kiernan said.
Because of the different treatment implications related to each type of dementia, Prof Kiernan said it’s crucial to be very specific in diagnosis.
“The key focus now is to come up with blood tests and the first of those blood tests was approved by the FDA a month ago. That blood test is to look for the p-tau protein [phosphorylated tau 217, associated with Alzheimer’s dementia], and that’s now being used by the TGA as a diagnostic test,” he said.
“The basis for the test came from British bio bank studies. They had 55,000 people that had been going to get MRI scans, bloods, all of these tests every year.
“Not just for dementia, for anything to do with medicine. After about 14 years, they found that around 1400 of that 55,000 had developed dementia, so they took all of the blood tests over the last 10 years, looking at every single protein in the blood.
“There are hundreds of thousands of proteins, but by putting the tests through AI, they found that there were four proteins in the blood that, if you combine them, basically guaranteed a diagnosis of Alzheimer’s disease.”
Most crucial of all? These proteins are elevated 10 years before symptoms of Alzheimer’s develop. And while we don’t yet have the infrastructure in Australia to be able to process these kinds of tests here, Prof Kiernan said it’s an inevitable future.
“Probably in about four or five years time, you’ll be referred from your GP to the local pathology service, you’ll get the blood test, and if the p-tau protein test is positive, that will be the diagnosis for Alzheimer’s disease,” he said.
Being able to make that diagnosis 10 years before symptoms set in allows for a whole new suite of treatments, preventive measures and research.
AI is revolutionising treatment and research
As well as being able to analyse massive amounts of data, predictive AI has been put to work diagnosing dementia as well.
A University of Cambridge research project built a machine learning model that was able to accurately predict (at a success rate of 81 per cent) the speed at which a person with mild cognitive impairment would develop Alzheimer’s, as well as which people would most likely remain stable. The AI model was more successful than current diagnostic models.
Another machine learning model out of the University of California can predict the disease up to seven years before symptoms appear.
Elsewhere in the research, important discoveries about links to other conditions or factors are being discovered, with dementia’s links to ADHD, hormones (particularly menopause) and even Herpes currently being explored.
And while there are still systemic, financial and political barriers and challenges involved with the great leaps forward his sector is making, Prof Kiernan agreed that this period is one of hope.
“I think the message is one of optimism and empowerment,” he said.
“And we’re not just talking about improving things for carers or making people comfortable at the end stages of their lives, we’re talking about improving outcomes and brain health for the entire community.”
Originally published as Aussie researchers at the forefront of massive dementia breakthroughs
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