Dr. Haggarty’s research focuses on gaining a fundamental understanding of the molecular and cellular mechanisms of neuroplasticity that enable the nervous system to sense, adapt, and respond to a variety of internal and external stimuli.

Discovering New Medicines for Brain Disorders Using Personalized Stem Cell Models

Stephen Haggarty, PhD
Stephen Haggarty, PhD
Stuart and Suzanne Steele MGH Research Scholar 2017-2022
Associate Investigator in Neuroscience, Massachusetts General Hospital
Associate Professor of Neurology, Harvard Medical School

Researchers face a number of challenges in developing new treatments for neurodegenerative and psychiatric diseases such as Alzheimer’s, frontotemporal dementia (FTD) and autism, including: •The diverse and complex causes of these diseases •The limited ability to replicate the progression of these diseases in laboratory models •The difficulty of obtaining living human neurons to study the early stages of disease and model its development over time

Our research seeks to identify and validate new treatment strategies with an innovative process in which skin cells that are taken from patient samples can be genetically reprogrammed into pluripotent stem cells (starter cells that can be used to create a variety of cell types). These stem cells can then be turned into genetically identical neurons that can be used to model disease progression in the lab and screen for new treatments.

We are also working to speed up the drug discovery process with new methods for large-scale drug screening, and are designing new chemical probes to gauge the effectiveness of potential treatments. By collaborating with clinical specialists in neurodegenerative and psychiatric diseases, we hope to translate our findings into new treatments and diagnostic tools.

Our ultimate goal is to advance precision medicine-based approaches for both common and rare neurodegenerative and psychiatric diseases.