Amyotrophic lateral sclerosis (ALS) is a late-onset, devastating brain and spinal cord disease characterized by rapid death of brain and spinal cord cells. This degeneration leads to muscle wasting, paralysis, difficulty swallowing, slurred speech, lung failure and finally to patient death within an average of 3 to 5 years from disease onset. Currently there are just two FDA-approved drugs Riluzole and Edaravone for treating ALS. Riluzole can only prolong ALS patient lifespan by about two to three months. Edaravone was only approved by the FDA (USA) in 2017. This approval was in fact based on the results of an ALS study group in Japan, where they found Edaravone showed efficacy in early-stage patients with a diagnosis of ALS, however treatment for those at a more advanced stage will require more research. In this project we will examine the effects of edaravone on neuronal stem cells, or unspecialised cells which have the ability to develop into many different types of cells in the brain and spinal cord system. These stem cells could potentially be used to replace the diseased cells of ALS. This experiment has never before been carried out on human neuronal stem cells, and the effects remain entirely unknown. Through the use of induced pluripotent stem cell (iPSC) technology, we will be able to convert any human cell (except reproductive cells) from an ALS patient into stem cells which will retain all the disease’s characteristics. We can then investigate ALS and edaravone outside of the human body itself.
