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Researchers Just Proposed a Radical New Way of Defining Alzheimer’s. Could It Lead to Better Drugs?

Alzheimer's disease has been a quagmire for drug developers. Andrew Brookes—Getty Images/Cultura RF

Alzheimer’s has long been one of the heaviest therapeutic albatrosses slung round drug developers’ necks. The memory-eating disease, expected to afflict 15 million Americans by 2060 (and tens of millions more around the world as life expectancy increases), has no cure; a new drug for the condition hasn’t been approved in well over a decade; initially promising experimental treatments seem to be failing with clockwork regularity; and there’s not even a definitive consensus on what, exactly, biopharma companies should focus on while developing Alzheimer’s medicines.

A major new proposal from top Alzheimer’s groups may, at least in part, help shift this landscape. The Alzheimer’s Association and the National Institute on Aging released the proposed research framework on Tuesday—and it aims to focus more on prevention rather than treating people once they’re already grappling with Alzheimer’s devastating effects.

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Here’s how the groups put it in their official release on the guidelines, dubbed the NIA-AA Research Framework: Towards a Biological Definition of Alzheimer’s Disease: “This evolution of the previous diagnostic criteria is in line with most chronic diseases that are defined biologically, with clinical symptoms being a… consequence….The goal of much of medicine is to identify and treat diseases prior to overt symptoms. The [NIA-AA Research] Framework is intended to provide a path forward to… prevention trials of Alzheimer’s disease among persons who are clinically asymptomatic.”

In plainer terms, the researchers are arguing for a preventive approach that’s based on changes in internal biology rather than the behavioral and physical symptoms which plague Alzheimer’s patients and are visible to the non-clinical world, including friends and family members. As the Alzheimer’s Association notes, until recently, “clinical trials were conducted where up to 30% of participants did not have the Alzheimer’s disease-related brain change targeted by the experimental drug.”

So what does that mean for drug makers, practically speaking, if this framework is officially adopted? For one, it would give them three specific biological markers to hone in on: The buildup of beta amyloid and tau proteins, which cause brain plaques associated with Alzheimer’s, and brain nerve cell death. Picking up on these potentially tell-tale signs early on may help biopharma firms recruit a wider swath of patients at risk for developing the downstream symptoms of Alzheimer’s and create more treatments that help prevent that kind of progression—or at least that’s what the hope is.

The new Alzheimer’s framework may not be much of a surprise given numerous, high-profile, late-stage clinical trial failures in the field by companies like Eli Lilly and Merck, and the decision by other companies (such as Pfizer) to back away from the risk-prone field.