Grant: Project Grant

Although amyotrophic lateral sclerosis (ALS) has been known as a major neurodegenerative disease for decades, very few drugs have been approved for the treatment of ALS. With the increasing incidence and prevalence of ALS, there is a great need to discover new ways to treat the disease. Current ALS drug strategies have minimal therapeutic effects and scope due to modest survival benefits and less-than-ideal drug properties, and there remains no cure for the disease. Edaravone remains one of the few FDA/Health Canada-approved ALS drugs, but suffers from many limitations, such as poor drug properties. Edaravone also suffers from poor patient compliance due to the need for daily i.v. infusions for 14 consecutive days in ALS patients every month, and is a less-than-ideal drug. We propose that changing the structure of edaravone so it can overcome its current limitations by improving the overall drug properties of this marketed ALS drug using 'the tricks of the trade'. Our strategy is to improve upon current therapeutics through the development of new and improved versions of edaravone that possess improved drug properties. To accomplish this goal, we plan to create new and improved versions of edaravone, and test the drug-like properties of these molecules in test-tubes (in vitro) and in ALS animal models (in vivo). Overall, this will allow us to determine and optimize the drug properties of our new ALS drug and allow us to develop an optimized drug candidate that is ready for advanced clinical trials.