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 diagnose and impossible to treat. Recent advances in research are now changing this outlook.
In the past two years, the U.S. Food and Drug Administration approved the first drugs that slow Alzheimer’s symptoms such as memory loss. Progress in gene editing, brain imaging, blood testing, and epidemiology is contributing to new treatments and possible prevention strategies.
“We’ve had thousands of failed clinical trials and made very little progress against this devastating disease for decades,” said Martin Kampmann, professor of biochemistry and biophysics at UC San Francisco. “But just over the last five or so years, we’ve hit an inflection point. We’re starting to get traction.”
Two FDA-approved drugs target amyloid protein deposits in the brain, delaying cognitive decline by up to seven months. However, these medications do not cure or reverse Alzheimer’s 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 Azheimer’s,” said Adam Boxer, neurology professor at UC San Francisco. “But this is just the first generation.” He compared their development to early HIV drugs that were initially limited but improved over time: “now we have very effective treatments for HIV,” Boxer said.
Boxer leads a five-year clinical trial funded by the National Institutes of Health that combines an anti-amyloid drug with therapies targeting tau protein—another factor in Alzheimer’s progression. “The thought is that a combination of an anti-amyloid and an anti-tau drug could have a much larger effect than either alone,” he said.
Researchers at UCLA have discovered a molecule that increases gamma oscillations—brain signals important for short-term memory—which may help restore cognitive function lost due to Alzheimer’s. Istvan Mody, professor of neurology and physiology at UCLA Health explained: “The hope with the anti-amyloid drugs was that with the removal of the plaques, the disease would be cured… Unfortunately, that hasn’t been the case. The removal of these toxic compounds still leaves an altered brain behind.” His team found that mice treated with this molecule regained memory abilities comparable to healthy mice: “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 also improving diagnosis speed and accessibility for Alzheimer’s patients. In May 2025, the FDA approved a blood test detecting tau and amyloid proteins associated with dementia; it is currently available only for those already showing symptoms. Research led by Hector M. González at UC San Diego identified three additional blood molecules linked to cognitive decline among more than 6,000 Latino participants in a nationwide study—a step toward earlier detection before symptoms arise.
Rachel Whitmer from UC Davis highlighted lifestyle factors’ impact on dementia risk: “The good news is, we now know that 45 percent of the overall population risk of dementia is accounted for by modifiable factors like midlife hypertension, low social engagement, untreated hearing loss or hyperlipidemia, or exposure to air pollution.” Whitmer led studies showing improvements in cardiometabolic health during adolescence or young adulthood benefit long-term brain health.
She also heads UC Davis’s involvement in a two-year clinical trial (U.S. POINTER study) where participants followed programs involving exercise, diet changes, mental activities and peer support sessions; both intensive intervention groups showed higher cognitive scores after completion.
CRISPR gene editing technology—originally developed at UC Berkeley—is being used by Martin Kampmann’s lab at UCSF to explore genetic triggers underlying Alzheimer’s progression by modifying genes within cultivated brain tissue samples derived from patient cells. This work has helped identify specific genetic states influencing microglia (the brain’s immune cells), opening new avenues for targeted therapies beyond current medications.
Kampmann stated: “I think in the next five or 10 years we’ll see that it’s a combination of therapies…that can really be effective” against dementia onset or progression.
The University of California system plays a central role in advancing Alzheimer’s research through its ten campuses and six academic health centers—including eight out of ten California Alzheimer’s Disease Centers statewide—and operates several NIH-funded research centers focused on neurodegenerative diseases.
