Alzheimer's gene therapy shows promise to prevent brain damage
New Delhi: A novel gene therapy for Alzheimer's disease, which showed promise to protect the brain from damage and preserve cognitive function, has renewed hope for millions of patients with the neurodegenerative disease.
Worldwide, approximately 57 million people live with dementia, and Alzheimer's disease is the most common cause, accounting for 60-70 per cent of cases.
Alzheimer's 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, said researchers from the University of California San Diego School of Medicine.
They noted that, 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 behaviour of brain cells themselves.
The study investigated the effect of hippocampal SynCav1 delivery in two distinct preclinical mice models.
The results, published in the journal Signal Transduction and Targeted Therapy, showed 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.
Further, the finding stated that compared to healthy mice of the same age, the treated mice also had a similar pattern of gene expression. This suggests that the treatment has the potential to alter the behaviour of diseased cells to restore them to a healthier state.
'While multiple newly FDA-approved treatments focus on targeting amyloid-beta clearance in Alzheimer's patients, the therapeutic value of SynCav1 lies in its ability to protect vulnerable neurons and augment cellular responses -- mechanisms that differ from currently approved therapies,' said the researchers in the paper.
Due to the multitude of neurotoxicity in the Alzheimer's brain, the team called for further studies to investigate SynCav1's therapeutic role when combined with amyloid-targeted drugs to enhance clinical outcomes.
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