Innovative Drug Candidate ALT001 Disrupts Viral Pathways Linked to Alzheimer’s Disease

A groundbreaking study led by researchers at Korea University College of Medicine has revealed a promising approach to tackling Alzheimer's disease, particularly its connection to viral infections such as herpes simplex virus type 1 (HSV-1). The research focuses on a novel compound, ALT001, which has demonstrated the ability to interfere with the viral mechanisms that may accelerate neurodegeneration.

Recent scientific investigations suggest that certain neurotropic viruses could play a role in the onset of Alzheimer's disease. Dr. Ok Sarah Shin, a prominent professor in the Department of Convergence Medicine, along with colleagues Dr. Soo-Jin Oh and Professor Jean-Ho Yun, explored how HSV-1 infection impacts brain immune cells called microglia. Using diverse experimental models—including mice, human-derived microglia, co-cultures of microglia and neurons, and brain organoids—they observed that HSV-1 hampers mitophagy, a vital process for removing damaged mitochondria, leading to compromised mitochondrial functions.

Furthermore, the virus was found to impair phagocytosis, the process by which microglia clear amyloid plaques—protein deposits characteristic of Alzheimer’s pathology. These findings illuminate how viral infections can contribute to neurodegeneration.

Importantly, the research team developed ALT001, a mitophagy enhancer, which effectively suppressed HSV-1 replication and mitigated neuroinflammatory responses. The compound also improved microglia’s capacity to clear amyloid proteins by restoring mitophagy functionality. Dr. Shin emphasized the significance of these results, stating that they demonstrate at a molecular level how viral infections may exacerbate neurodegenerative diseases and highlight new avenues for therapeutic development.

This study, published in Theranostics, offers a promising outlook for future treatments targeting viral influences in Alzheimer’s disease and other neurological disorders caused by viral infections. The findings suggest that enhancing cellular cleanup processes in the brain could be a crucial strategy for combatting virus-related neurodegeneration.