Dr. Maragakis is Professor of Neurology and Director of the ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins, and the Co-Director of the Johns Hopkins ALS Clinic. In his clinical practice Dr. Maragakis treats patients with motor neuron diseases in a multidisciplinary environment where advancements made in the laboratory can be applied to treatment and disease management. These interventions include both multicenter clinical trials in ALS as well as investigator-initiated studies exploring the potential role of resistance and endurance exercise in ALS management.
Through the ALS Center for Cell Therapy and Regeneration Research, Dr. Maragakis’ focus is on applying what is learned from discoveries in disease biology to their translation into clinical interventions for ALS patients, principally through the targeting of cell therapeutics for ALS treatment. Much of his efforts have centered on discovering the role of astrocytes – the supporting cells of the brain – in causing and propagating ALS. His work in this area has led to the creation of lines of stem cells from ALS patients that can be used to advance the understanding of ALS astrocyte and motor neuron biology, and eventually help develop drug screening methods for ALS therapeutics. By transplanting glial stem cells into ALS animal models, the researchers in Dr. Maragakis lab found that these cells can engraft, migrate and differentiate into astrocytes, and subsequently provide neuroprotection to motor neurons that are otherwise vulnerable in patients with ALS. Under Dr. Maragakis’ direction his laboratory has amassed a large library of stem cells derived from patients with ALS. This collection is an unparalleled resource for the development of a translational research program, supporting the development of assays that can be used for both the basic understanding of ALS astrocyte and motor neuron biology as well as, eventually, drug screening for ALS therapeutics.