Dr. Nicholas Maragakis is the Director of the ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins as the Co-Director of the Johns Hopkins ALS Clinic.
Nicholas Maragakis treats patients with motor neuron diseases, such as amyotrophic lateral sclerosis (ALS). He serves as medical director of the ALS Clinical Trials Unit, an extension of the multidisciplinary clinic that seeks to facilitate opportunities for patients with ALS to participate in clinical trials that could advance the treatment of this disease.
Dr. Maragakis is interested in the basic science of understanding neurodegenerative diseases, as well as the translational potential of therapeutic compounds developed in the laboratory. His fundamental research interest is in using stem cell-derived motor neurons and glia — for understanding the development and propagation of ALS.
His laboratory has been involved in creating and characterizing lines of stem cells from patients with ALS using induced pluripotent stem cell (iPSC) methodologies. The laboratory has a large library of iPSCs from patients with familial ALS and from patients with sporadic ALS. The utilization of this library of iPSCs has facilitated the development of a spinal cord-specific iPSC-astrocyte/motor neuron co-culture system, which has been leveraged for 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.
His laboratory has taken its long-standing interests in astrocyte biology, as it relates to ALS, to develop a program that investigates astrocyte-specific mechanisms as contributors to the progression (both temporally and anatomically) of disease in ALS. In this vein, there is an interest in astrocytic glutamate transporters and, more recently, astrocyte hemichannels and gap junctions.
The second major line of investigation utilizes the understanding about astrocytes and stem cells to develop a translational program developing glial restricted progenitor cells for transplantation into ALS models and, more recently, the potential for transplantation into patients with ALS. The lab is focused on applying their understanding of the disease biology in the laboratory to the translation of these discoveries for ALS patients.