Alzheimer’s wonder drug: Once-a-day pill could halt disease

The drug is designed to target the brain’s release of stress hormone cortisol, recognised as a key driver of neurodegeneration.

Actinogen Medical CEO Dr Steve Gourlay

Six Queenslanders are taking part in an international clinical trial for what’s being billed as a potential holy grail treatment for Alzheimer’s disease, with more participants being sought.

The trial of the drug, Emestedastat, is being conducted by Sydney-based biotech company Actinogen Medical, which has spent the last decade developing the pill originally discovered by researchers at the University of Edinburgh.

The drug, which goes by the brand name Xanamem, is designed to target the brain’s release of stress hormone cortisol, recognised as a key driver of neurodegeneration.

The World Health Organisation this year gave Xanamem a first-in-class, which Actinogen said was a rare global endorsement that placed Actinogen as one of the most promising players in neuroscience worldwide.

Actinogen CEO Steve Gourlay told The Courier-Mail: “This could just be the holy grail for Alzheimer’s disease, or at least part of the holy grail as a combination therapy.

“We’re the only ones in the world developing a drug like this that can get into the brain and do what we hope will significantly slow Alzheimer’s disease.”

Dr Gourlay said the molecule for Xanamem was developed by a “very smart chemist” at the University of Edinburgh to penetrate the brain and reduce cortisol levels.

“The idea is that you need your stress response in times of when you need to be defensive or run away from a threat, but putting your brain in a chronic state of stress is bad for you,” he said.

“For example, if you take cortisol as a pill, say for asthma or arthritis – if you take that for a few weeks, you get depressed, you get muscle wasting, you get diabetes.

“All sorts of terrible things happen.

“And so the whole idea is that chronically elevated levels of cortisol in the brain essentially are bad for you, toxic to nerve cells and cause inflammation.”

Dr Gourlay said Xanamem was designed to be a once-a-day pill to treat one of the underlying causes of Alzheimer’s, unlike new treatments targeting amyloid plaques and requiring costly monthly infusions.

Jim Northey, pictured with wife Bev, is taking part in the trial. Picture: Lachie Millard

“We’re not trying to mop up the protein that forms as a result of Alzheimer’s disease (causing) inflammation and nerve damage, but we’re actually trying to treat the underlying biology of the disease,” he said.

He said about 400 people had been treated with the medication so far, with promising results in slowing the progression of Alzheimer’s, as well treating chronic depression.

About 220 Alzheimer’s sufferers in Australia and the US are taking part in a clinical trial that Actinogen hopes could see the drug receive approval from Australia’s Therapeutic Goods Administration and America’s Food and Drug Administration.

Queensland trials are being run by the University of Sunshine Coast and Prince Charles Hospital, with participants including 81-year-old Caloundra man Jim Northey.

Mr Northey’s wife Bev said she had noticed significant improvement in her husband during the treatment, to the extent she believed he must have been given Xanamem rather than the placebo which some trialists receive.

“Initially, I didn’t know if it was going to work but as we got going along, we could see some definite improvements in Jim,” she said.

“He was much more engaging, he was confident in himself, his memory in the tests they gave him … I could see some real improvements in him. I felt very confident he was either not getting any worse or certainly getting better.

“We weren’t looking for a cure, but we thought it might slow things down. It showed so much promise”

Mrs Northey said since finishing the 12-month trial about six weeks ago and resuming his old medication, her husband had regressed.

“He’s got quieter in himself, he doesn’t want to go places,” she said.

“He certainly has slipped backwards, not massively, but it’s noticeable.”

Dr Gourlay said it could be the end of the decade before Xanamem was publicly available.

“Developing a novel drug for Alzheimer’s disease is quite a complicated and time-intensive business,” he said, adding that more Queensland participants were being sought for the trial.

Mrs Northey said: “It might not do much for us, but maybe it will be something that will help our children down the track.”