Background

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a debilitating disease characterized by rapidly progressive weakness, muscle atrophy and spasticity, and difficulty speaking, swallowing, and breathing. Worldwide, ALS kills about 100,000 people and 125,000 new cases are diagnosed per year. Apart from Riluzole, and recently Edaravone (the only two drugs approved by the FDA for treatment of ALS), researchers have been largely unsuccessful at translating promising results from pre-clinical research to effective pharmacotherapies for ALS patients. One of the problems in finding highly efficacious treatments in ALS is believed to derive from the issue of disease-driven pharmacoresistance mediated by ABC drug efflux transporters. These transporters are commonly found in the blood-brain barrier and play a crucial