Unlike current Alzheimer’s treatments that focus on clearing harmful protein build-up in the brain, this new gene therapy targets the root of the disease by directly modifying how brain cells function.

Alzheimer’s disease, which affects millions globally, is driven by the accumulation of abnormal proteins in the brain that ultimately kill brain cells and impair memory and cognition. Existing treatments help manage symptoms, but the new gene therapy aims to stop or even reverse the disease’s progression.

In a study using mice, researchers administered the gene therapy during the symptomatic stage of the disease. The treated mice retained memory functions dependent on the hippocampus a key region affected in Alzheimer’s. Additionally, their gene expression patterns resembled those of healthy mice, suggesting the therapy may reprogram diseased brain cells toward a healthier state.

Though more research is needed before human clinical trials can begin, this gene therapy represents a promising strategy for slowing cognitive decline and supporting brain health.

The findings were published in Signal Transduction and Targeted Therapy. The study was led by senior author Dr. Brian Head, professor of anesthesiology at the UC San Diego School of Medicine and a research scientist at Veterans Affairs, along with co-senior author Dr. Shanshan Wang, assistant professor of anesthesiology at UC San Diego. The therapy was licensed by UC San Diego to Eikonoklastes Therapeutics in 2021. The company also received FDA Orphan Drug Designation for its use in treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

Source: https://www.universityofcalifornia.edu/news/alzheimers-gene-therapy-shows-promise-preserving-cognitive-function