Brain Program
PARTNERS
Department of Neurosurgery
Department of Neurology
Department of Biomedical Sciences
FACULTY LEADERSHIP
Robert Baloh, MD, PhD
Tyler Pierson, MD, PhD
Clive Svendsen, PhD
Terrence Town, PhD
OVERVIEW
Diseases of the nervous system cause misery for millions of people around the world. At the Regenerative Medicine Institute (RMI), scientists and clinicians are exploring how diseases of the brain occur and how we may be able to protect or regenerate the brain by using combinations of drugs and cells.
THE NEED
As people live longer, diseases of the brain become more prevalent. For example, 50 percent of people over the age of 80 will develop Alzheimer’s disease. There are very few treatments available for any diseases of the brain or spinal cord, and novel approaches are desperately needed.
THE CHALLENGE
We have to better understand what causes diseases of the brain and how they progress. The brain is enormously complex, making repair difficult. Cells and drugs have difficulty getting into the brain from the blood, and there are few good animal models of neurological disorders.
OUR APPROACH
We are working with adult skin cells from patients with neurological diseases and taking the cells back in time to a pluripotent state. These cells are then used to create “disease in the dish” models where new neurons undergo degeneration similar to that seen in the patient. In cell therapy studies, similar cells are then modified to release drugs and then placed back in the brain to see if they can repair function lost from neurological diseases. We are also exploring adult stem cells and the interaction between the immune system and the brain and how this may impact neurological diseases such as Alzheimer’s. Finally, we are trying to understand how proteins build up in cells that die in neurological disorders and why they are so destructive. We have generated pluripotent stem cells patients with ALS (Lou Gehrig’s disease), Down syndrome, spinal muscular atrophy, and Huntington’s disease and are using them to model these disorders.
ON THE HORIZON
We are moving toward a clinical trial with the Department of Neurosurgery using novel neurosurgical methods and stem cells secreting powerful growth factors to treat ALS.
Baloh Lab
Svendsen Lab
Town Lab
