Research Team Successfully Targets Common Mutation in ALS and Dementia

Scientists from The Scripps Research Institute (TSRI) and the Mayo Clinic have for the first time successfully designed a therapeutic strategy targeting a specific genetic mutation that causes a common form of amytrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, in addition to a type of frontotemporal dementia (FTD). The research team developed small-molecule drug candidates and showed they interfere with the synthesis of an abnormal protein that plays a key role in causing both diseases. Additionally, the team developed biomarkers that can test the efficacy of this and other therapies. “Our small molecules target a genetic defect that is by far the most major cause of familial ALS, and if you have this defect you are assured of getting ALS or FTD,” said the study’s lead author. Currently, ALS is usually fatal two to five years after diagnosis, and there is no effective treatment for FTD, a neurodegenerative disease that destroys neurons in the frontal lobes of the brain. To read more about this study, click here.