Neurodegenerative Diseases

LSP investigators are developing new approaches for detecting and treating neurodegenerative diseases like Alzheimer's Disease. 

As populations age, neurodegenerative diseases like Alzheimer's disease and ALS pose an overwhelming burden for healthcare systems and tragedies for the individuals and families involved. Unfortunately, it remains challenging to discover effective drugs for neurodegenerative diseases because their causes are complicated and poorly understood. In part, this is because neurodegenerative diseases often develop gradually over years before the disease becomes symptomatic, so the disease is advanced at the time symptomatic patients present to healthcare providers.

LSP researchers are collaborating with clinicians at Mass General Brigham to improve how we understand, detect, and treat neurodegenerative diseases. Led by Dr. Mark Albers, these efforts evaluate potential therapeutic targets in various preclinical models (cultured human cells, iPSCs, mouse models, and human tissues), using mechanistically-informed biomarkers for early detection, drug response prediction, and monitoring efficacy in clinical trials. Additional projects leverage machine learning algorithms (DRIAD: Drug Repurposing In Alzheimer's Disease; TRIALS (Therapeutic Repurposing in ALS) to explore opportunities for repurposing FDA-approved drugs for new indications. Our long-term goal is to better understand disease mechanisms and guide the development of molecules with companion biomarkers tailored to specific types of Alzheimer's Disease and other age-related neurodegenerative diseases.