Health Check: Researchers find new ways to navigate the Alzheimer’s disease treatment maze
An offshore trial has found a link between kidney malfunction and Alzheimer’s, while a local biotech legend is taking a different route.
A global study links Alzheimer’s disease with kidney malfunction
FDA czar Dr Marty Makary outlines the agency’s priorities
Pro Medicus fesses up to a cyber breach
The quest for an Alzheimer’s disease cure has taken a new twist, with an offshore clinical study linking impaired kidney function with the malady.
As reported in Neurology, people with impaired kidney function have higher levels of Alzheimer’s biomarkers in their blood.
A Swedish study enrolled 2,279 people with an average age of 72. These candidates did not have dementia at the start of the trial.
Participants had medical exams and took tests to measure cognitive skills.
They also had blood tests to measure kidney function and levels of Alzheimer’s disease biomarkers, such as tau and amyloid beta proteins.
“Our study found that when the kidneys are not functioning properly, there may be higher levels of Alzheimer’s biomarkers in the blood,” said study author Francesca Gasparini, of Stockholm’s Karolinska Institutet.
She said reduced kidney function per se did not increase the risk of developing dementia.
But “we did find that impaired kidney function may accelerate the onset of dementia in people who have higher levels of biomarkers”.
This “highlights the need for doctors to consider kidney function when interpreting results of Alzheimer’s biomarkers in the blood.”
Local biotech legend takes a different tack on Alzheimer’s
Meanwhile, the Herald Sun reports that Australian IVF pioneer Professor Alan Trounson will launch a company to develop the world’s first nasal spray for Alzheimer’s and other neuro-inflammatory conditions.
The early-stage therapy is based on clearing the amyloid plaque buildup in the brain. This is done by stimulating the immune system.
Amyloid protein build-up is a known factor with Alzheimer’s.
But Trounson says “there’s nobody else effectively taking this direct immune repair approach at present".
The therapy is based on harvesting extracellular vesicles from natural killer cells – the blood’s powerful immune cells.
The new private company is called Evinco Therapeutics.
Actinogen girds for late-stage trial results
Also meanwhile, Actinogen Medical (ASX:ACW) is in phase IIb/III stage of its Xanamia trial.
The study tests the nexus between excess brain cortisol levels and Alzheimer's.
The double-blinded, placebo-controlled study has enrolled 220 patients with mild to moderate Alzheimer’s.
This is determined by “clinical criteria and confirmed by an elevated level of the pTau181 protein biomarker in blood”.
The company expects the trial to be fully enrolled by the end of 2025, with initial interim results due in late January next year.
Actinogen expects final topline results in late 2026.
The company believes its lead compound Xanamem also could be relevant for Fragile X syndrome and “other neurological and psychiatric diseases”.
Actinogen’s phase IIa treatment-resistant depression trial, Xanacidd, also “showed clinically and statistically significant benefits”.
Marty Makary’s musings from the Mount
The FDA’s new commissioner Dr Marty Makary has cast light on the agency's priorities in 2026 – and his comments provide succour to several ASX biotechs.
Addressing Citi's Global Healthcare Conference in Miami, Florida, the RFK Junior appointee nominated universal and/or longer-lasting flu vaccines.
This is relevant for CSL (ASX:CSL), which has been buffeted by the Trump administration’s anti-jab stance.
Makary also wants to see expedited development of promising cancer drugs. This could be relevant for Telix Pharmaceuticals' (ASX:TLX) ambition to move from radiodiagnostics to radiotherapies.
He also called out the need for new treatments for neurodegenerative disease and post-traumatic stress disorder.
Neurodegenerative disease exponents include the aforementioned Actinogen, Alterity Therapeutics (ASX:ATH) (motor system atrophy) and Neuren Pharmaceuticals (ASX:NEU).
Having commercialised a Rett syndrome drug, Neuren has advanced programs for the same-but-different Phelan-McDermid, Pitt Hopkins and Angelman syndromes.
Makary also suggested that late-stage clinical trial requirements could be modernised to reduce trial approval times.
Vaunted measures include reducing the standard requirement for two randomised controlled phase III trials to one.
Makary also reassured the nervous biotech industry that the FDA’s recent budget cuts were “administrative” and did not affect scientific reviewers or inspectors.
He also introduced the long-awaited Commissioner's National Priority Voucher program, which should quicken review timelines for worthy drug candidates.
Cynata gets stem-cell trial safety tick
A local clinical trial safety gatekeeper has cleared stem cell developer Cynata Therapeutics (ASX:CYP) to go to the next stage of its kidney transplant trial.
The Data Safety Monitoring Board (DSMB) found no safety concerns with the first cohort of the study. This means the company can proceed to – drum roll – cohort two.
The trial appraises Cynata’s Cymerus mesenchymal stem cell therapy for kidney transplant recipients. This is an alternative to relying on long term immunosuppressant drugs.
A leader in kidney transplants, The Netherlands' Leiden University Medical Centre is managing and funding the phase I/II effort.
The first cohort received a single intravenous infusion of CYP-001 six weeks after a kidney transplant, in addition to standard treatment.
The DSMB found no episodes of kidney transplant rejection, or serious adverse events.
Cynata chief medical officer Dr Jolanta Airey says the program aims to enshrine CYP-001 treatment as a "safe and efficacious" alternative to current anti-rejection drugs.
“While these established drugs are quite effective in preventing rejection, they are associated with significant toxicity.”
Cynata is also undertaking a phase III trial for osteoarthritis and a phase II effort for steroid-resistant acute graft-versus-host disease.
The company also has completed a phase I diabetic foot ulcer study.
Pro Medicus says cyber breach was an isolated event
Pro Medicus (ASX:PME) says “speculation” about an isolated cyber security incident was, in fact, grounded in fact.
The radiology imaging giant says an unknown party accessed a single email inbox in July this year.
The miscreant “potentially could have accessed” personally identifiable information for around 100 current and former Pro Medicus employees.
The perpetrator could not access client systems or patient data.
While Pro Medicus did not incur any financial loss or “operational impact”, the snafu blots the copybook on the company's otherwise stellar year marked by a string of client wins.
The episode also shows how entities that sell products based on secure IT need to be on their guard – always.
But – hey – we’re not saying anything new.
At Stockhead, we tell it like it is. While Alterity and Cynata are Stockhead clients, the companies did not sponsor this article.
Originally published as Health Check: Researchers find new ways to navigate the Alzheimer’s disease treatment maze
