Researchers at Washington University School of Medicine have developed an experimental immunotherapy they say could change how Alzheimer’s disease is treated — and potentially delayed — particularly for people at highest genetic risk.
The approach targets amyloid beta, a sticky protein that builds into nerve-damaging plaques in the brain and is widely believed to play a major role in Alzheimer’s, especially in early-onset cases. In tests on mice, researchers say a single injection of the engineered therapy cut amyloid plaques in half, raising the possibility of slowing — or altering — the course of the disease.
The findings are early and have not yet been proven in humans. But they build on a growing body of research suggesting that removing amyloid early and consistently may delay the onset of symptoms.
WashU researchers reported in Lancet Neurology that a subset of 22 participants who received amyloid-removing drugs for an average of eight years saw their risk of symptom onset cut roughly in half.
David M. Holtzman, a professor of neurology at WashU Medicine and co-author of a paper on the new treatment, said timing appears critical.
“Consistent with the antibody drug treatments, this new CAR-astrocyte immunotherapy is more effective when given in the earlier stages of the disease,” Holtzman said. “But where it differs, and where it could make a difference in clinical care, is in the single injection that successfully reduced the amount of harmful brain proteins in mice.”
The experimental therapy draws on advances in cancer treatment known as CAR-T, which engineers a patient’s cells to target disease. In this case, researchers reprogram astrocytes — brain cells — to act as amyloid-clearing agents, an approach described as turning them into “amyloid-gobbling machines.”
Even as researchers point to the promise of the approach, they caution that significant work remains to optimize the treatment, understand potential side effects and translate the findings into safe, effective therapies for people. Similar approaches in other diseases have shown only modest progress and, in some cases, raised safety concerns.
The urgency is clear. According to the Centers for Disease Control and Prevention, nearly 7 million Americans are living with Alzheimer’s in 2026, a number expected to double by 2060.
For patients with inherited forms of the disease, the stakes are especially high.
Jake Heinrichs, a New York City resident and participant in a Washington University Alzheimer’s clinical trial since 2012, has spent much of his life expecting the disease to follow a familiar and devastating path through his family.
He suffers from Autosomal Dominant Alzheimer’s disease, a rare, inherited form that typically causes symptoms to appear before age 60, often in a person’s 40s or 50s.
Alzheimer’s is not just a research subject for Heinrichs; it is encoded in his DNA. His father, grandmother, uncle and older brother all died from complications related to the disease, often at similar ages.
Heinrichs began showing symptoms at 40.
“I remember feeling helpless and him not being able to comprehend what I was asking him … and I just started crying,” he said of watching his father decline as a child.
Now 51, Heinrichs has lived more than a decade beyond the point when his family history suggested he would already be in steep decline. He remains symptom-free, something he attributes in part to his participation in clinical trials targeting amyloid.
“It’s still a study but it has given me an extension to my life that I never banked on having,” Heinrichs told The Associated Press.
Recent treatments that remove amyloid through regular infusions have already shown they can slow cognitive decline by about 30% over 18 months in patients with early symptoms, according to Dr. Jon LaPook, chief medical correspondent for CBS News.
For Heinrichs, those advances have made possible decisions he once thought unlikely. After more than a decade receiving amyloid-targeting treatments, his condition has remained stable long enough for him and his wife, Rachel Chavkin, a Broadway director, to have a child.
“They now have this beautiful kid, Sam … I mean, what a moment for them,” LaPook said in a televised segment.
In an earlier interview with the Associated Press, Heinrichs said he hopes his son will be spared the experience that defined his own childhood — watching a parent slowly fade.
Rachel Chavkin said the potential of these treatments is measured not just in years, but in quality of life.
“It’s just about keeping people alive or helping them live better,” she said. “And, in this case, it’s helping my husband survive.”
Sylvester Brown Jr. is the Deaconess Foundation Community Advocacy Fellow.