Alzheimer’s disease affects about 10 percent of people over age 65 and is a leading cause of death, surpassing both breast cancer and prostate cancer. For many years, the disease was difficult to predict or diagnose and had no effective treatments.
Recent advances in science may be changing this situation. In the last two years, the FDA approved drugs that can delay symptoms such as memory loss. Progress in areas like gene editing, brain imaging, blood testing, and epidemiology is contributing to new possibilities for treatment and prevention.
“We’ve had thousands of failed clinical trials and made very little progress against this devastating disease for decades,” said Martin Kampmann, professor at UC San Francisco who studies dementia at the molecular level. “But just over the last five or so years, we’ve hit an inflection point. We’re starting to get traction.”
The first drugs approved by the FDA target amyloid protein deposits in the brain linked with Alzheimer’s. These drugs can delay cognitive decline by up to seven months but do not cure or reverse the disease and have notable side effects.
“There is concern from some that these medicines are not as effective or as safe as desired for people with symptoms of Alzheimer’s,” said Adam Boxer, neurology professor at UC San Francisco. “But this is just the first generation.” He compared these medications to early HIV treatments that initially offered limited benefits but eventually led to highly effective therapies after more research. Boxer leads a five-year NIH-funded clinical trial combining anti-amyloid medication with experimental tau-targeting therapies: “The thought is that a combination of an anti-amyloid and an anti-tau drug could have a much larger effect than either alone.”
Researchers at UCLA have identified a molecule that boosts gamma oscillations—brain signals associated with memory—which may help restore cognitive function in Alzheimer’s patients. Istvan Mody, professor at UCLA Health, explained: “The hope with the anti-amyloid drugs was that with the removal of plaques, the disease would be cured… Unfortunately, that hasn’t been the case.” His team found their compound improved maze navigation in mice models: “We’ve shown that this works in mice,” Mody said. “If we can develop a therapy that’s safe and effective for people, we may be able to restore cognitive function. That’s the ultimate hope.”
Blood tests are now making diagnosis more accessible after recent FDA approval for detecting tau and amyloid proteins related to Alzheimer’s through blood samples rather than more invasive procedures. This test is currently only approved for symptomatic patients; research continues into earlier detection markers.
A study led by UC San Diego neuroscientist Hector M. González identified three additional molecules in blood samples from over 6,000 Latino participants correlated with cognitive decline. One marker (NfL) showed promise as an early indicator even before symptoms appear.
Rachel Whitmer from UC Davis emphasized lifestyle factors’ impact on dementia risk: “We now know that 45 percent of overall population risk of dementia is accounted for by modifiable factors,” she said—listing midlife hypertension, low social engagement, untreated hearing loss or high cholesterol among them—and highlighted research showing improvement when targeting multiple health behaviors simultaneously.
On genetic fronts, Martin Kampmann’s lab uses CRISPR technology—developed at UC Berkeley—to edit genes in cultured brain tissue derived from patient cells to study how different gene states affect brain health outcomes such as inflammation or neuronal survival versus damage: “I think in the next five or 10 years we’ll see it’s a combination of therapies…that can really be effective” against dementia onset.
Across its ten campuses and six academic health centers—including eight out of California’s ten Alzheimer’s Disease Centers—the University of California plays a significant role in advancing Alzheimer’s research and care nationwide.
However, federal funding cuts being considered by Congress could threaten ongoing momentum in university-based scientific innovation across America.
