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Engineered molecules carrying Alzheimer’s therapies to the brain.
Fluorescent dye highlighting dementia-inducing proteins.
Brain cells growing in laboratory dishes.
It’s all part of a wave of cuttingedge research exploring new ways to treat and potentially provide a cure for Alzheimer’s disease and other forms of dementia.
At universities and laboratories across the globe, thousands of research projects representing billions of dollars in public and private funding are underway with the goal of understanding the complex diseases hidden deep inside the brain.
Some studies are looking for new therapies and medications. Others are searching for ways to predict and diagnose Alzheimer’s before symptoms even begin. Some are investigating how diet and sleep can affect the development of the disease.
Myriad projects mark what many physicians and scientists consider a golden age of Alzheimer’s research that offers exciting promises for those with the disease, according to Rebecca Edelmayer, the Alzheimer’s Association’s senior director of scientific engagement.
“I think we’re really in a new era with this field in terms of how we understand the disease, how we can diagnose individuals and detect the disease at its earliest stages,” she said.
Those promises started to be fulfilled in July when the Food and Drug Administration fully approved Leqembi, a drug many have heralded as the first to treat the underlying causes of the disease and not just its symptoms. Studies show the medication modestly slows the disease in its early stages.
That’s a win, but it’s a far cry from the kind of relief many living with dementia are seeking, according to Steve Steffensen, associate professor of neurology at the Dell Medical School at the University of Texas at Austin.
“They’re good medications, but they’re not great medications, and they do not cure the disease,” he said. “They can prolong independence, but typically it’s on the order of months, not years.”
DRUG TRIAL
Finding a cure, or at least a better treatment, is something Myra Garcia, of Upland, California, hopes she can help make happen.
The 65-year-old was once an opera singer and university fundraiser. Now, she’s in a clinical trial for an up-and-coming drug called donanemab, developed by Indianapolis-based Eli Lilly.
In May, the company released its most current testing results, which found nearly half of participants had no clinical progression of Alzheimer’s at the first year. The drug also resulted in 40% less decline on the ability to perform daily living activities. That’s the strongest data yet for a treatment.
Garcia has participated in the drug trial for around two years at the University of Southern California after she was diagnosed with Alzheimer’s in 2020. She receives an infusion and medication once a month.
“I think it’s working,” Garcia said. “It could be the placebo effect, but I feel like I’m doing really well.”
The medication, like Leqembi, works by removing buildup of dementia-associated plaques in the brain. But clinical trials for donanemab are also testing its effectiveness at fighting another brain protein called tau, which can cause cognitive decline.
Tau can twist into tiny fibers called tangles, which cause vital brain cell transport systems to collapse. The Lilly tests found “meaningful positive results” for those with intermediate to high levels of tau.
The drug is in phase 3 testing. Eli Lilly plans to apply for FDA approval of donanemab this year.
Garcia said she’s grateful to be part of a drug trial that could lead to a treatment that vastly improves the lives of those living with the disease. Had she been diagnosed just 20 years ago, nothing close to these medications would exist, she noted.
“I’m part of the treatment generation, and I’m so happy and so pleased to be a part of that,” Garcia said. “I think it is just an amazing point in time.”
ONE-AND-DONE PILL UNLIKELY
The recent success and fast pace of Alzheimer’s research has many suffering from dementia hoping for a cure.
Most clinicians and scientists believe that, as with cancer and heart disease, there won’t be a one-and-done pill that reverses the disease, Edelmayer said. It will more likely entail a combination of powerful drugs and risk reduction strategies.
Treatment research today not only focuses on the brain but also includes investigating how the heart, blood vessels and hormones affect brain function and health.
That’s something Anne Robinson, head of Carnegie Mellon University’s chemical engineering department, found in her research. Studies Robinson conducted in 2022 point to the advantages of a holistic approach to treating the disease rather than finding a silver-bullet cure.
“Based on our findings, Alzheimer’s requires a similar approach to HIV treatment, where you have a cocktail of drugs used to address different elements of the disease,” she said. “Together, these drugs work toward the larger goal: treating the symptoms of Alzheimer’s in a particular patient.”
No cure may exist yet, but the sheer number of studies and research topics underway provides optimism to scientists and patients alike. The National Institutes of Health this year alone is providing $3.8 billion in research funding — the most ever allocated.
For Garcia, it all points to a brighter future for those like her struggling with Alzheimer’s.
“It’s wonderful that there are so many scientists working on this,” she said. “Sometimes they hit the mark and sometimes they don’t, but I think that all of it is just so very important.”
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