Repurposing drugs to treat Alzheimer’s Disease

LSP investigators working with the Massachusetts Alzheimer's Disease Research Center at the MGH are developing new approaches to treat Alzheimer's Disease based on the hypothesis that the disease has multiple distinct etiologies, some involving degeneration-associated chronic inflammation.

As populations age, Alzheimer's disease poses a rapidly increasing burden for healthcare systems, and the disease is a tragedy for the individuals and families involved. Discovering new drugs for neurodegenerative diseases is challenging because their cause is poorly understood, they progress relatively slowly, and end-stage disease – when symptoms are most obvious - remains difficult to treat. For these reasons, we are pursuing multiple approaches to repurposing FDA-approved drugs for other indications. To investigate repurposing opportunities, we are mining large-scale clinical databases (in collaboration with colleagues in the UK) and developing pre-clinical models of neurodegeneration. The machine learning algorithm DRIAD (Drug Repurposing In Alzheimer's Disease), for example, uses profiling data from human neural cell cultures treated with existing compounds to generate ranked lists of possible repurposing candidates. Some of these drug candidates have advanced to early-stage clinical trials. Our long-term goal is to use repurposed drugs to better-understand disease mechanisms and guide the development of more effective molecules that are tailored to specific types of Alzheimer's Disease.