Mitigated Motor Function Impairment by Enhanced Delivery of Edaravone via Ultrasound with Microbubbles in a Transgenic Mice Model of Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a prototypical neurodegenerative disease featured by progressive degeneration and extensive loss of upper and lower motor neurons. As a free radical scavenger, edaravone (EDA) has been proven to exert neuroprotective effects. In this study, the delivery of EDA was enhanced by low-intensity focused ultrasound combined with microbubbles (FUS/MB+EDA) in a transgenic ALS mice model. The frontal cortex/plasma ratio of EDA concentration after FUS/MB+EDA treatment increased by 2.7 folds of the EDA only group. The motor function evaluation tests revealed that FUS/MB+EDA treatment induced more effective mitigating effect on the motor function impairment of the ALS mice than the EDA only group, which was validated by histology of motor neurons in the ventral horns of spinal cords. The preliminary results of this study suggested that FUS/MB facilitated the delivery of EDA into the brain across the blood-brain barrier and effectively exerted positive effects on slowing down the progress of motor functional deficits in the transgenic ALS mice.