The core group of investigators at the Ansari ALS Center brings a unique perspective to ALS therapeutics with specific expertise in working with patients with ALS and other motor neuron diseases, as well as basic science and clinical research interests and programs for this disease.
Nicholas J. Maragakis, M.D., Director
Dr. Maragakis, is an Associate Professor in the Department of Neurology at Johns Hopkins and Co-Director of the ALS Clinic. He has both basic science and clinical research interests. His basic science interests include the role of astrocytes in neurodegenerative diseases: specifically Amyotrophic Lateral Sclerosis (ALS). His laboratory is specifically focused on the role of glial progenitor responses during the course of motor neuron degeneration and how mutant SOD1 influences glial stem cell differentiation and ultimately astrocyte function. Most recently, interests have included attempts to understand how astrocytes in ALS may help to propagate disease spread both temporally and anatomically. In collaboration with Johns Hopkins neuroscientists, current efforts are underway to create lines of stem cells from ALS patients using induced pluripotent stem cell (iPSC) methodologies. This will allow for the development of human cell lines which can be used for both the basic understanding of ALS astrocyte and motor neuron biology, and eventually, drug screening for ALS therapeutics. These studies are all being performed with an eye towards translational applications. The laboratory has also focused on the potential role of astrocyte replacement in ALS using glial stem cells called glial restricted precursors. Current efforts are underway to design a human clinical trial using human glial restricted precursors. His clinical research interests have included both multicenter clinical trials in ALS as well as investigator-initiated studies investigating the potential role of resistance and endurance exercise in ALS management.
Dr. Maragakis' Recent Research
- Focal Transplantation–Based Astrocyte Replacement is Neuroprotective in a Model of Motor Neuron Disease
- Human Glial-Restricted Progenitor Transplantation into Cervical Spinal Cord of the SOD1G93A Mouse Model of ALS
- Astrocytes carrying the superoxide dismutase1 (SOD1G93A) mutation induce wild-type motor neuron degeneration in vivo