Researchers
at University of California San Diego School of Medicine have developed
a gene therapy for Alzheimer’s disease that could help protect the
brain from damage and preserve cognitive function.
Unlike existing treatments for Alzheimer’s that target unhealthy
protein deposits in the brain, the new approach could help address the
root cause of Alzheimer’s disease by influencing the behavior of brain
cells themselves.
Alzheimer’s disease affects millions of people around the world and
occurs when abnormal proteins build up in the brain, leading to the
death of brain cells and declines in cognitive function and memory.
While current treatments can manage symptoms of Alzheimer’s, the new
gene therapy aims to halt or even reverse disease progression.
Studying mice, the researchers found that delivering the treatment at
the symptomatic stage of the disease preserved hippocampal-dependent
memory, a critical aspect of cognitive function that is often impaired
in Alzheimer’s patients. Compared to healthy mice of the same age, the
treated mice also had a similar pattern of gene expression, suggesting
that the treatment has the potential to alter the behavior of diseased
cells to restore them to a healthier state.
While further studies will be required to translate these findings
into human clinical trials, the gene therapy offers a unique and
promising approach to mitigating cognitive decline and promoting brain
health.
The study, published in Signal Transduction and Targeted Therapy,
was led by senior author Brian Head, Ph.D., professor of anesthesiology
at UC San Diego School of Medicine and Veterans Affairs research career
scientist, and co-senior author Shanshan Wang, M.D. Ph.D., an assistant
professor of anesthesiology at UC San Diego School of Medicine. The
gene therapy technology was licensed by UC San Diego to Eikonoklastes
Therapeutics in 2021. Eikonoklastes was granted Orphan Drug Designation
(ODD) by the FDA for the use of the patented gene therapy in amyotrophic
lateral sclerosis (ALS) also known as Lou Gehrig’s disease.
