A copper-based drug helped the brain flush out Alzheimer’s toxins, cutting plaque buildup and improving memory in a promising new study.
Scientists at Monash University have identified a promising new approach to tackling Alzheimer’s disease. In laboratory studies, a copper-delivering drug significantly lowered levels of toxic proteins linked to the disease while also improving long-term spatial memory.
The findings, published in ACS Chemical Neuroscience, suggest the compound Cu(ATSM) may help restore an important brain cleanup process that breaks down in Alzheimer’s disease. Researchers say the treatment repairs a key waste removal pump at the blood-brain barrier, opening the door to a potential new strategy for addressing neurovascular dysfunction associated with the condition.
Repairing the Brain’s Waste Removal System
Alzheimer’s disease is characterized by the accumulation of amyloid-beta, a toxic protein that builds up in the brain. Under normal conditions, these proteins are transported out of the brain and into the bloodstream through the blood-brain barrier.
A major part of that process relies on specialized transport proteins known as P-glycoprotein (P-gp) pumps. In people with Alzheimer’s disease, these pumps become less effective, allowing harmful proteins to accumulate inside the brain.
Lead author Dr. Jae Pyun from the Drug Delivery, Disposition and Dynamics theme at the Monash Institute of Pharmaceutical Sciences (MIPS) said the treatment works by engaging the brain’s blood vessels to help reduce toxic protein levels, leading to measurable benefits in behavior and cognition.
“This is the first study to show that Cu(ATSM) can increase the abundance of P-gp clearance pumps in an Alzheimer’s model, by 24.1 percent, effectively linking the repair of the blood-brain barrier to a reduction in toxic proteins and improved cognitive function,” Dr. Pyun said.
“By improving the pumps, the brain can finally clear out the trapped waste. Over 56 days, the treatment reduced toxic amyloid-beta by 42 percent and improved spatial learning by nearly 44 percent.”
Potential Path Toward Human Trials
Senior author Professor Joseph Nicolazzo, Director of the Centre for Drug Candidate Optimisation at MIPS, said the drug could move relatively quickly toward clinical testing for Alzheimer’s because it has already undergone safety assessments for other neurological conditions.
“Cu(ATSM) is a copper compound with anti-inflammatory and neuroprotective properties that has already progressed to clinical testing for conditions like Parkinson’s and ALS,” Professor Nicolazzo said.
“Because reducing amyloid burden is clinically proven to improve functional outcomes, these preclinical results strongly support the rationale for testing this drug in early symptomatic Alzheimer’s disease.”
How Does the Brain Clear the Proteins?
Although the researchers observed a substantial reduction in amyloid buildup, they are still investigating the exact pathways that allow these proteins to leave the brain.
In addition to repairing the blood-brain barrier, the team believes the copper treatment may also stimulate microglia, the brain’s resident immune cells. These cells could help break down and remove the toxic plaques that contribute to Alzheimer’s disease.
Future research will focus on identifying the precise mechanisms responsible for clearing amyloid-beta from the brain into the bloodstream. The current results provide strong support for further investigation of biometal therapies such as Cu(ATSM) as potential treatments for blood vessel dysfunction and memory decline associated with Alzheimer’s.
Growing Need for New Alzheimer’s Treatments
Alzheimer’s disease and other forms of dementia continue to pose a major global health challenge. In Australia, dementia recently became the nation’s leading cause of death, surpassing coronary heart disease.
With populations aging and dementia related deaths continuing to rise, researchers emphasize the urgent need for therapies that can slow or prevent cognitive decline.
Reference: “Cu(ATSM) Restores Blood–Brain Barrier Abundance of P-Glycoprotein and Improves Cognitive Function in the APP/PS1 Mouse Model of Alzheimer’s Disease” by Jae Pyun, Asif Noor, Pranav Runwal, Celeste Mawal, Oliver K. Fuller, Casey L. Egan, Mark A. Febbraio, Paul S. Donnelly, Jennifer L. Short, Ashley I. Bush and Joseph A. Nicolazzo, 30 May 2026, ACS Chemical Neuroscience.
DOI: 10.1021/acschemneuro.6c00252
The study was led by Dr. Jae Pyun and included co-authors Pranav Runwal, Oliver Fuller, Casey Egan, Professor Mark Febbraio, Associate Professor Jennifer Short, and Professor Joseph Nicolazzo from the Monash Institute of Pharmaceutical Sciences. Collaborators also included Dr Asif Noor, Celeste Mawal, Professor Paul Donnelly, and Professor Ashley Bush from the University of Melbourne.
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