Novel Compounds with Neuroprotective Potential for Amyotrophic Lateral Sclerosis (ALS)

Title: Novel Compounds with Neuroprotective Potential for Amyotrophic Lateral Sclerosis (ALS)  Invention: The present invention is a series of compounds developed at the University of Arizona that may be used in the treatment of ALS, particularly in treating the locomotor dysfunction associated with the disease. The compounds have a neuroprotective effect in that they prevent the aggregation of TDP-43, a neurotoxic protein associated with neurological disorders including ALS.  Background: Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that causes muscle weakness, disability, and eventually death, with a median survival rate of three to five years. TAR DNA-binding protein 43 (TDP-43), encoded by the TARDBP gene, is the major disease protein in ALS. TDP-43 aggregation is linked with frontotemporal lobar degeneration and ALS; some neuroprotective drugs focus on the TDP-43 aggregation pathway as a means of treating ALS. Applications:ALS therapeutics; reduction of locomotor dysfunction associated with ALSTreatment of other neurological disorders associated with TDP-43 and TARDBP dysfunction Advantages:Focuses on an under-targeted pathway Licensing Manager:Rakhi GibbonsRakhiG@TLA.arizona.edu(520) 626-6695

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