New gene therapy targets the root cause of Alzheimer’s disease

In a development that could redefine Alzheimer’s disease treatment, scientists at the University of California, San Diego School of Medicine have unveiled a new gene therapy designed to prevent brain damage and safeguard cognitive function. 

Unlike current medications that merely manage symptoms or target protein build-up, researchers say this approach intervenes at the cellular level, potentially halting or reversing the disease’s progression.

Alzheimer’s disease, which affects millions of people around the world, is characterized by the accumulation of abnormal proteins in the brain that ultimately kill neurons, leading to memory loss and cognitive decline. The gene therapy seeks to prevent this by influencing the behavior of brain cells, rather than just clearing harmful proteins.

The therapy was tested in mice that had already developed Alzheimer’s symptoms. Following treatment, the mice showed significant improvements in memory, particularly in functions tied to the hippocampus, the part of the brain crucial for learning and memory. Even more compelling was the fact that treated mice exhibited gene expression patterns resembling those of healthy mice, indicating that diseased cells were being restored to a healthier state.

The findings, published in the journal Signal Transduction and Targeted Therapy, offer early evidence that the therapy could serve as a truly disease-modifying intervention.

What’s next?

“Our results demonstrate that it’s possible to alter the trajectory of Alzheimer’s by targeting the biology of the brain’s own cells,” said senior author Dr. Brian Head, a professor of anesthesiology and research scientist with the Veterans Affairs system.

Co-senior author Dr. Shanshan Wang, an assistant professor at UC San Diego School of Medicine, echoed the potential of this innovation: “This could open the door not only for Alzheimer’s treatment but also for addressing other neurodegenerative conditions.”

UC San Diego licensed the gene therapy technology to biotech firm Eikonoklastes Therapeutics in 2021. The FDA has already granted Orphan Drug Designation to Eikonoklastes for using the same platform to treat amyotrophic lateral sclerosis (ALS), further underscoring its therapeutic potential.

While human clinical trials are still on the horizon, this study represents a significant shift in the Alzheimer’s treatment landscape. If successful in people, the therapy could move beyond managing symptoms to genuinely modifying the disease itself—bringing new hope to patients and families around the world.