FDA starts a program to help test ways to prevent Alzheimer's

The US Food and Drug Administration has set up a new program to deal with the scientific and regulatory problems that slow down the development of treatments for brain disorders. Its first project focuses on one of the hardest goals in medicine: preventing or delaying Alzheimer’s disease.¹

The program was announced on October 22 and is called the Neuroscience Advancement and Innovation Nexus, or NAIN. It is meant to coordinate expertise across the agency’s centers that review medical products.¹ There is no new drug, no new study result, and no change to any approval decision in this announcement. It is a plan for how the agency will work on the problem.

What the FDA announced

NAIN brings together experts from several parts of the FDA, including the Center for Drug Evaluation and Research, which reviews most medicines, and the Center for Biologics Evaluation and Research, which handles products such as vaccines and many gene and cell therapies.¹

Its first effort is named the Alzheimer’s Prevention Accelerator. According to Medical Daily’s report, the accelerator will explore ways to improve clinical trials (the studies in people that test whether a treatment helps). Two questions are at the center: how researchers choose who takes part, and how they measure whether a treatment delays cognitive decline, meaning the slow loss of memory and thinking skills.¹

The scale of the problem is large. The Alzheimer’s Association’s 2026 Facts and Figures report estimates that 7.4 million US adults aged 65 and older are living with Alzheimer’s dementia this year.¹

Why prevention studies are so difficult

Alzheimer’s builds up over many years. To test whether a drug or other approach prevents it, researchers have to decide whether it works before clear symptoms appear.¹ That creates practical problems. A study may need to run for a long time. Many volunteers may never get sick during the study, whether or not they get the treatment. And the early changes researchers want to slow can be hard to measure.

These problems are not new. In 2018, when the FDA issued draft guidance on early Alzheimer’s, BioPharma Dive reported that people in the earliest stage are particularly hard to study because they have no thinking or physical symptoms even as the brain changes typical of the disease begin.² Drugmakers have focused on this group anyway, because researchers believe stopping damage at this stage is key to preventing later decline.²

The 2018 guidance said that results on biomarkers (lab or scan measures that track the disease, rather than how a person feels or functions) could be enough to win a faster “accelerated” approval in very early disease with few or no symptoms. But the agency also noted there was no agreement on which biomarkers should be used to judge whether a treatment is having an effect.² Questions about how to pick participants and how to measure a slowdown are the same ones the new accelerator says it will explore.¹

What the program does not do

Medical Daily’s report is direct on this point: the initiative does not introduce a new Alzheimer’s prevention drug and does not guarantee that researchers will develop one.¹ Its stated aim is to address barriers that make brain research difficult.¹

Several things are not yet clear from the available reporting:

• which specific trial rules or methods, if any, will change;
• whether the program will produce new written guidance for companies and researchers, and when;
• how outside groups such as patient organizations, universities or drug companies will take part;
• which brain disorders beyond Alzheimer’s the program will take on next.

Biohack’s sources do not include the FDA’s own announcement, so details beyond those in the news report are not covered here.

How the FDA has handled Alzheimer’s trials before

The FDA has adjusted its approach to Alzheimer’s studies several times, often after pressure from the field or a run of failures.

2010-2011: loosening safety exclusions

In 2010, after brain scans of trial volunteers showed small bleeds and swelling linked to drugs that target amyloid (a protein that forms plaques in Alzheimer’s brains), the FDA tightened its research guidelines. It excluded people with more than two existing tiny brain bleeds and stopped testing in people who developed bleeds or swelling.³ A work group of scientists from drug companies, academic centers and the Alzheimer’s Association argued these limits came before the data showed they were needed. In 2011 the association announced that the FDA had accepted its recommendations to change the rules.³

“We’ve gone from concept to consensus to publication in less than 1 year, reflecting the urgent need to move the field forward.”

That was Maria Carrillo, then a senior director at the Alzheimer’s Association, as quoted by Psychiatric Times.³

2018: guidance on very early disease

In February 2018 the FDA issued five guidance documents to support drug development for Alzheimer’s and other neurological conditions. The Alzheimer’s draft updated guidance from 2013 and set out the agency’s thinking on who to enroll and what to measure in trials of early disease.² Then-Commissioner Scott Gottlieb wrote that the document described approaches for studying people who had no visible impairment but could be identified through sensitive memory testing, imaging or biomarkers.²

At the time, BioPharma Dive noted that a handful of drugs eased Alzheimer’s symptoms but none changed the course of the disease, and that industry efforts had produced a long list of setbacks.² That same week Merck stopped a late-stage study of verubecestat in very early Alzheimer’s, and earlier that month Boehringer Ingelheim ended work on another candidate after it missed its goals in a mid-stage trial.²

How it fits the wider field

Research on catching and slowing Alzheimer’s early is active across labs and universities. Northwestern University, for example, has reported animal work in which an experimental drug showed promise as an early intervention before symptoms began, and a study suggesting an older approved anti-seizure drug can stop plaques from forming in models of the disease.⁴ These are early lab and animal findings, and they show the kind of prevention ideas that would eventually need human trials of the sort the FDA program is meant to make easier to run.

Better ways to measure early decline are also being studied. Biohack recently covered work on short voice recordings that estimate a “speech age”, one of several attempts to track brain changes earlier. Whether any such measures end up being accepted in trials is the kind of question regulators weigh.

Medical Daily’s coverage appeared alongside its report on a new US Department of Health and Human Services dementia plan focused on prevention and early detection, which dropped an expired 2025 treatment goal.¹ How the two efforts relate is not described in the sources available to Biohack.

What to watch next

• Concrete outputs. Whether the Alzheimer’s Prevention Accelerator produces meetings, draft guidance or proposed changes to how trial volunteers are chosen and how decline is measured.¹
• The biomarker question. The FDA flagged in 2018 that there was no agreement on which biomarkers should be used in early disease.² Any new position on this would matter to companies planning prevention trials.
• Next disorders. NAIN covers brain disorders broadly, and Alzheimer’s is only its first project.¹
• Effect on real trials. The test of the program will be whether prevention studies become faster, smaller or clearer to judge. That will take years to see.

For now, the news is an organizational step at the FDA. It names a problem many researchers have described for over a decade and assigns a team to it. It does not change what is known about any treatment.