$250,000 awarded to Dr. Sahara Khademullah at the Cervo Brain Research Centre, Université Laval.
Within the brain and spinal cord, neurons pass electrical signals to each other through specialized chemicals called neurotransmitters, capable of either exciting or inhibiting them. Maintaining a proper balance between the excitatory and inhibitory signals is crucial for normal brain function and allows for complex movements like walking.
In ALS, there is an early imbalance between excitation and inhibition in the nervous system. This imbalance may be linked to changes in the inhibitory system, and modifying this system has shown promise as a treatment strategy for ALS. With this award, Dr. Khademullah will investigate the effectiveness of using gene-based therapies to restore a normal balance in mouse models of ALS.
The study will focus on a protein called KCC2, which acts like a gatekeeper controlling the entry and exit of chloride ions from cells as part of the inhibition process. Dr. Khademullah has previously shown that KCC2 levels are decreased in ALS mice prior to the onset of symptoms, leading her to believe that restoring normal levels of KCC2 may help to correct the excitation imbalance and slow or even stop disease progression. The findings from this study will provide early preclinical evidence for gene-based strategies that, if successful, could pave the way for further development of innovative treatments for people living with ALS.
Dr. Khademullah is committed to applying her training and expertise to ALS research, aiming to validate KCC2 as a therapeutic target for ALS. Ultimately, her goal is to establish a research group investigating the role of inhibition and excitation in ALS, contributing to advancements in understanding and treating the disease.