A compound that ferries copper into the brain cut levels of toxic Alzheimer's proteins and improved long-term spatial memory in laboratory experiments, according to Monash University researchers writing in ACS Chemical Neuroscience. The drug, Cu(ATSM), appears to work by repairing a specific piece of plumbing at the blood-brain barrier rather than by attacking amyloid plaques directly.
That plumbing matters. Amyloid-beta, the sticky protein central to Alzheimer's, is normally pushed out of the brain and into the bloodstream across the blood-brain barrier by transport pumps known as P-glycoprotein, or P-gp. In Alzheimer's, those pumps weaken considerably, and the drain effectively clogs - toxic protein that should be leaving the brain stays put. The Monash work suggests Cu(ATSM) restores that pump's function.
Lead author Dr Jae Pyun of the Drug Delivery, Disposition and Dynamics theme at the Monash Institute of Pharmaceutical Sciences, for whom the study capped a PhD project, said the treatment engaged the brain's blood vessels to lower toxic protein levels, and that this translated into behavioural benefits - in this case, better long-term spatial memory.
The practical interest here is the target. Most high-profile Alzheimer's drugs aim at amyloid itself; this approach goes after neurovascular dysfunction, the failure of the brain's blood vessels and clearance systems, which opens a different therapeutic route. These are laboratory findings, so the next question is whether the same pump-repairing effect holds up in humans.