U.K. scientists have charted a new path in attacking Alzheimer's disease, concocting an oral formulation that halted brain tissue death in mice and giving hope for a fresh weapon against biotech's memory-robbing white whale.
In a study on mice with prion disease, a team from the U.K.'s Medical Research Council found that their oral inhibitor of the kinase PERK completely stopped neurodegeneration, making it the first compound to do so on any living brain.
The inhibitor--initially developed by GlaxoSmithKline ($GSK) as a cancer treatment--helps the brain cope with broken proteins generated by Alzheimer's, Parkinson's and Huntington's diseases. The brain's natural response to each is shutting down protein production, but that only spurs cell death and accelerates neurodegeneration. However, by targeting PERK, researchers were able to keep brain tissue intact, forging a new pathway for future shots at treating brain disease.
"This finding, I suspect, will be judged by history as a turning point in the search for medicines to control and prevent Alzheimer's disease," King's College London's Roger Morris told the BBC.
As with any promising animal study, the results need years of follow-up before the compound will be ready for work in the exponentially more complicated brains of human beings, but the investigators' repeated uses of "landmark," "turning point" and "dramatic" aren't entirely unwarranted.
To date, most Alzheimer's research has focused on targets unique to the disease--whether beta-amyloid, tau or other proteins--and while that work has amounted to some intriguing therapies, the world's drug developers have failed left and right to cobble together effective treatments, enduring a string of late-stage disasters for once-promising drugs.
But by targeting what is common between all neurodegenerative diseases, the council's compound suggests a single treatment that could halt the process of memory loss and cell death. And, considering it's the first chemical to effectively stop neurodegeneration, it's easy to understand all the enthusiasm.
"The world won't change tomorrow," Morris said, "but this is a landmark study."
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