$125,000 awarded to Dr. Chantelle Sephton, Université Laval, in collaboration with Dr. Liang Li, University of Alberta.
Lipids encompass a diverse group of molecules, including fatty acids, oils, and specific components of cell membranes. They play crucial roles in the body, serving as a source of energy, contributing to the construction of cell membranes, and participating in signaling pathways. The cellular processes responsible for managing these lipids are collectively referred to as lipid metabolism. Recent studies suggest that dysregulation of these processes may occur in ALS, leading to the accumulation of lipid droplets in brain cells.
With this award, Dr. Sephton will investigate how lipid metabolism is affected in ALS, utilizing a mouse model carrying a mutation in the ALS-associated gene fused in sarcoma (FUS). These ALS-FUS mice, validated in the Sephton lab, display both motor function and cognition defects, similar to those commonly observed in ALS. Additionally, they exhibit the accumulation of lipid droplets and toxic lipid byproducts in specific brain regions, making them an ideal model for studying how lipid metabolism is affected in ALS. These investigations will also extend to other ALS mouse models, including SOD1 and TDP-43. Furthermore, researchers will explore whether the drug arimoclomol can influence lipid dysregulation in these diverse models.
By examining the broader role of lipid metabolism in ALS, this study will help to deepen our understanding of its significance to the disease. The ultimate goal is to identify potential new targets for intervention, providing valuable insights that could pave the way for effective therapeutic strategies, not only in ALS but also in other neurodegenerative diseases.