People suffering from the early onset of Alzheimer’s disease are being given access to a revolutionary drug that’s being hailed as a potential cure.
The drug, Xanamem, works by blocking the production of intracellular cortisol through the inhibition of the 11β-HSD1 enzyme in the brain.
Taken in a tablet, scientists at Actinogen Medical – the company behind the drug – have described the drug as ‘exercise in a pill’.
Dementia affected an estimated 55.2million people globally in 2020, 60–70 percent of whom had Alzheimer’s disease.
In tandem with increasing longevity, recent studies suggest the number is anticipated to grow to 139million by 2050, with the greatest increases in low and middle income countries.
It has been long reported that chronically elevated cortisol levels in the blood and the brain are associated with cognitive impairment.
Actinogen CEO Doctor Steven Gourlay told Daily Mail Australia a trial is underway across Australia and the United States with 220 participants.
The trial will evaluate Xanamem’s efficacy in Alzheimer’s patients with mild to moderate conditions.
Participants will take a 10mg daily dose for 36 weeks, with clinical endpoints assessing cognitive and functional outcomes.
A positron emission tomography (PET) scan of the human brain
The results from a previous trial have already indicated the drug could be ‘the Holy Grail’ of Alzheimer’s treatments.
History shows most people only get tested for Alzheimer’s when they notice a decline in their short-term memory.
‘People start to play puzzles and do things that hopefully keeps their mind fit, but exercise has been proven as the best method of delaying Alzheimer’s,’ Dr Gourlay said.
‘But the Holy Grail for Alzheimer’s is if people notice significant early short-term memory loss they can go to a memory clinic or a specialist, get diagnosed and start taking safe and effective easy-to-use therapy that will essentially stop that from progressing completely.
‘In other words, the hope is that these patients with early Alzheimer’s can potentially live a healthy, stable life. It’s like some cancers are now completely treatable and HIV is completely treatable and people die of old age with HIV.
‘The hope is Alzheimer’s can be like that.’
While that world does not currently exist, Dr Gourlay said the new drug is close to proving it can deliver just that.
While a drug for Alzheimer’s has been marketed for 20 years as a ‘memory booster’, the effects are not long term and have various side effects.
New drugs that see patients undergo intravenous medications every few weeks are known to swell the brain and slow the progression very slightly, Dr Gourlay said.
‘In fact when doctors are using these medications they can’t tell whether the medication is working because it’s such a mild effect,’ he said.
Actinogen CEO Doctor Steven Gourlay believes his scientists have created the ‘Holy Grail’ of Alzheimer’s treatment
Xanamem is a once-a-day pill that has already been safely tested in more than 400 people.
‘What we saw in an analysis of patients with progressive, mild Alzheimer’s disease is a really big effect to slow progression than anything that’s ever been seen before with any other medication,’ Dr Gourlay said.
That result needs to be confirmed before the pill go can to market, which is why a new trial is now underway.
The trial will run for nine months and compare the results with participants that take the active drug and those provided with a sugar pill.
‘Half will get the drug and half with get the placebo. The doctors and the patients don’t know which one is which and we do lots of assessments to measure their baseline abilities and we measure that every three months roughly over the course of the clinical trial to make sure that we can understand whether or not our drug is significantly slowing progression compared to the placebo group,’ Dr Gourlay said.
The company has already seen a dramatic slowing of that progression during previous testing of the same patients – albeit in tests over a shorter period and in a smaller number of participants.
‘Essentially this drug has a unique mechanism of action, it controls the stress hormone cortisol that is known to be strongly associated with Alzheimer’s and with progression and by doing that it really has the distinct possibility of being, if not the Holy Grail, at least a big part of the Holy Grail,’ Dr Gourlay said.
The awful effect of Alzheimer’s on the human brain
Scientists hope the pill will further help people already trapped in the nightmare of full blown Alzheimer’s.
‘We are testing in patients with both mild and moderate Alzheimer’s. By the time you get to moderate Alzheimer’s you need a fair bit of home support from your family or professional carers or looking at a nursing home,’ Dr Gourlay said.
‘It’s too early to say whether the drug will be as effective in the moderate or the mild, but we are investigating that and the hope is it will work for both.’
Dr Gourlay said the brain has the capacity to regenerate.
‘We like to think this drug is a cure, meaning patients can go back to normal memory, but I think that’s a bit optimistic. I think the Holy Grail here is to stop progression in its tracks and then allow people to spend their elderly years independently at home,’ he said.
The drug could even prove helpful for people not suffering from the onset of dementia, such as ordinary boozehounds.
‘You can get dementia from too much booze, I’m afraid to tell you,’ Dr Gourlay said.
‘This drug probably does help to prevent brain cell loss to a certain extent, but the simple answer is there are probably better things you could do on the boozehound front than take this drug.’
Should the latest trial be successful as expected, Dr Gourlay said the pill could go to market as early as 2027 if approved by regulators as a matter of priority.
Once on the market, it is predicted ordinary people would start undergoing genetic testing in anticipation of identifying a gene linked to Alzheimer’s with a view to jumping on the pill as soon as possible.
‘There’s no point testing for Alzheimer’s if there’s no treatment, but the hope is you will be able to do that and if you are at risk you have screening blood test, like you might screen for bowel cancer, and when that starts to go up you maybe have some brain scans and start the treatment,’ Dr Gourlay said.
People can join the trial here.
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