Frontotemporal Dementia (FTD) is the most frequently occurring dementia after Alzheimer's Disease (AD) with an expected increasing prevalence due to the aging population. A GGGGCC repeat expansion in C9ORF72 is the most common cause of this disease, accounting for about 10% of each disease worldwide. Unfortunately, at current, there are no effective therapeutic strategies available for either FTD. This proposal is focused on the development of a novel therapeutic strategy for FTD by testing a novel lead compound in relevant animal models as a preclinical proof-of-concept. Our lead compound was identified as highly neuroprotective in a high throughput small molecule screening using patient-derived induced neurons in our laboratory.