Introduction to Alzheimer’s Research
An experimental treatment appears to delay Alzheimer’s symptoms in some people genetically destined to get the disease in their 40s or 50s, according to new findings from ongoing research now caught up in Trump administration funding delays. The early results — a scientific first — were published even as study participants worried that politics could cut their access to a possible lifeline.
The Study’s Objective
The research led by Washington University in St. Louis involves families that pass down rare gene mutations almost guaranteeing they’ll develop symptoms at the same age their affected relatives did – information that helps scientists tell if treatments are having any effect. The new findings center on a subset of 22 participants who received amyloid-removing drugs the longest, on average eight years. Long-term amyloid removal cut in half their risk of symptom onset, researchers reported in the journal Lancet Neurology.
The Importance of the Study
Despite the study’s small size, “it’s incredibly important,” said Northwestern University neuroscientist David Gate, who wasn’t involved with the research. Now participants have been switched from an earlier experimental drug to Leqembi, an IV treatment approved in the U.S., to try to answer the obvious next question. “What we want to determine over the next five years is how strong is the protection,” said Washington University’s Dr. Randall Bateman, who directs the Dominantly Inherited Alzheimer’s Network of studies involving families with these rare genes.
Jake Heinrichs prepares for his infusion treatment with an experimental anti-amyloid Alzheimer’s drug while at home in New York, on Wednesday, March 12, 2025. (AP Photo/Heather Khalifa)
Funding Delays and Concerns
Here’s the worry: Bateman raised money to start that confirmatory study while seeking National Institutes of Health funding for the full project but his grant has been delayed as required reviews were canceled. It’s one example of how millions of dollars in research have been stalled as NIH grapples with funding restrictions and mass firings. At the same time researchers wonder if NIH will shift focus away from amyloid research after comments by Dr. Jay Bhattacharya, nominated as the agency’s new director.
The Science Behind Alzheimer’s
Scientists don’t know exactly what causes Alzheimer’s, a mind-destroying disease that affects nearly 7 million Americans, mostly late in life. What’s clear is that silent changes occur in the brain at least two decades before the first symptoms — and that sticky amyloid is a major contributor. At some point amyloid buildup appears to trigger a protein named tau to begin killing neurons, which drives cognitive decline. Jake Heinrichs sits with his 3-year-old son, Sam, during his infusion treatment with an experimental anti-amyloid Alzheimer’s drug in New York, on Wednesday, March 12, 2025. (AP Photo/Heather Khalifa)
Future Research Directions
Tau-fighting drugs now are being tested. Researchers also are studying other factors including inflammation, the brain’s immune cells and certain viruses. NIH’s focus expanded as researchers found more potential culprits. In 2013, NIH’s National Institute on Aging funded 14 trials of possible Alzheimer’s drugs, over a third targeting amyloid. By last fall, there were 68 drug trials and about 18% targeted amyloid.
Conclusion
Northwestern’s Gate counts himself among scientists who “think amyloid isn’t everything,” but said nothing has invalidated the amyloid hypothesis. He recently used brain tissue preserved from an old amyloid study to learn how immune cells called microglia can clear those plaques and then switch to helping the brain heal, possible clues for improving today’s modest therapies. For now, amyloid clearly is implicated somehow and families with Alzheimer’s-causing genes are helping answer a critical question for anyone at risk: Can blocking amyloid buildup really stave off symptoms? Without NIH funding, Bateman said, that opportunity will be lost. Jake Heinrichs looks through old family photos with his son, Sam, on Wednesday, March 12, 2025, in New York. (AP Photo/Heather Khalifa)
FAQs
Q: What is the current state of Alzheimer’s research?
A: Researchers have found a hint at how to delay Alzheimer’s symptoms, but they need to prove it through further studies.
Q: What is the role of amyloid in Alzheimer’s disease?
A: Amyloid is a major contributor to Alzheimer’s disease, and researchers are studying its role in the disease.
Q: How many people are affected by Alzheimer’s disease in the US?
A: Nearly 7 million Americans are affected by Alzheimer’s disease, mostly late in life.
Q: What is the goal of the current study on Alzheimer’s disease?
A: The goal is to determine if blocking amyloid buildup can really stave off symptoms of Alzheimer’s disease.
Q: Why is NIH funding important for Alzheimer’s research?
A: NIH funding is crucial for continuing the research and finding a cure for Alzheimer’s disease.
Originally Published: March 20, 2025 at 2:33 PM EDT
