For years now the gold standard for R&D in Alzheimer’s disease has focused on generating convincing evidence that any new therapy being studied could slow the cognitive decline of patients and help preserve their ability to perform the kind of daily functions that can keep a patient independent for a longer period of time.
That’s a hurdle no one has managed to clear for well over a decade. So now, with late-stage clinical failures piling up, the U.S. Food and Drug Administration (FDA) has set off down a path to adapt those standards as researchers are pushed inexorably into earlier and earlier forms of the disease, ahead of the brain damage inflicted by Alzheimer’s.
In a set of draft guidances, the agency essentially proposed to offer an approval pathway for new drugs that could prevent the onset of the devastating symptoms of Alzheimer’s if drug developers could hit acceptable biomarkers that indicate the drug is working. And they’re likely going to continue with a new gold standard that will focus on long-term cognition alone, lowering the bar for drugs for an enormous and growing market.
David Miller, the clinical vice president of Bracket, a Washington, D.C.-based tech provider which specializes in Alzheimer’s studies, tells me the draft guidance hit just after a meeting of the Washington, D.C.-based Alzheimer’s Association research group, which was discussing how you might be able to use a mix of markers for amyloid β and tau—two toxic proteins frequently cited as likely triggers—alongside neurodegenerative markers to identify patients who could be enrolled at a very early point in the disease.
“It’s ahead of where it was,” Miller says about their understanding of presymptomatic biomarkers. “There’s been an improvement in our understanding of how these biomarkers work together, where there might be improvement. That doesn’t mean we are where we need to be, but we are getting closer.”
“We need to figure out ways to do measurements better, sensitive to earlier stages of the disease, looking at cognition and function for more sensitive ways of doing it,” Miller says.