Nicholas John Maragakis, M.D.

Headshot of Nicholas John Maragakis
  • Director, Center for ALS Specialtiy Care
  • Professor of Neurology


Amyotrophic Lateral Sclerosis (ALS), Lou Gehrig's Disease, Neurology more

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The Johns Hopkins Hospital (Main Entrance)

Appointment Phone: 410-614-9874
1800 Orleans St.
Sheikh Zayed Tower
Baltimore, MD 21287
The Johns Hopkins Hospital (Main Entrance) - Google Maps

Johns Hopkins Outpatient Center (now called Levi Watkins, Jr., M.D., Outpatient Center)

Appointment Phone: 410-614-9874
601 N. Caroline St.
Baltimore, MD 21287
Johns Hopkins Outpatient Center (now called Levi Watkins, Jr., M.D., Outpatient Center) - Google Maps


Nicholas Maragakis treats patients with motor neuron diseases, such as amyotrophic lateral sclerosis (ALS). This care is coordinated with the Johns Hopkins Center for ALS Specialty Care, a multidisciplinary clinic with expertise in treating patients with ALS and providing support to their caregivers. 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 cells — more specifically, 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. more


  • Director, Center for ALS Specialtiy Care
  • Director, ALS Center for Cell Therapy and Regeneration Research
  • Medical Director, ALS Clinical Trials Unit
  • Professor of Neurology

Departments / Divisions

Centers & Institutes



  • MD; The University of Utah (1994)


  • Neurology; Johns Hopkins University School of Medicine (1998)


  • Clinical Neurophysiology; Johns Hopkins University School of Medicine (1999)

Board Certifications

  • American Board of Psychiatry And Neurology (Neurology) (2000)

Research & Publications


The Maragakis lab's main focus is in understanding disease mechanisms and targeting cell therapeutics for Amyotrophic Lateral Sclerosis (ALS). In collaboration with Johns Hopkins neuroscientists, his laboratory helped create stem cell lines from ALS patients using induced pluripotent stem cell (iPSC) methodologies. These cells 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, as well as eventually identifying ALS therapeutics. Researchers in the Maragakis lab also focus on the potential therapeutic role of astrocyte replacement in ALS using glial stem cells. By transplanting glial stem cells into ALS animal models, the researchers in the Maragakis lab found that the stem cells can engraft, migrate and differentiate into astrocytes, and subsequently provide neuroprotection to vulnerable motor neuron pools. The team uses stem cell transplantation biology to understand the influences of mutant SOD1 astrocytes on normal, healthy motor neurons. A significant laboratory effort is underway to translate these discoveries into therapies for patients with ALS.

Lab Website: ALS Center

Selected Publications

View all on PubMed

Taga A, Dastgheyb R, Habela C, Joseph J, Richard J-P, Gross SK, Lauria G, Lee G, Haughey N, Maragakis NJ.  Role of human induced pluripotent stem cell-derived spinal cord astrocytes in the functional maturation of motor neurons in a multielectrode array system.  Stem Cells Transl Med. 2019 Dec;8(12):1272-1285. doi: 10.1002/sctm.19-0147. Epub 2019 Oct 21 PMID:31631575 PMCID:PMC6877769

Almad AA, Doreswamy A, Gross SK, Richard JP, Huo Y, Haughey N, Maragakis NJ.  Glia. 2016 Apr 16. doi: 10.1002/glia.22989. Connexin 43 in astrocytes contributes to motor neuron toxicity in amyotrophic lateral sclerosis.

Haidet-Phillips AM, Doreswamy A, Gross SK, Tang X, Campanelli JT, Maragakis NJ. Human glial progenitor engraftment and gene expression is independent of the ALS environment. Exp Neurol. 2015 Feb;264:188-99. PubMed PMID: 25523812. 

Haidet-Phillips AM, Roybon L, Gross SK, Tuteja A, Donnelly CJ, Richard JP, Ko M, Sherman A, Eggan K, Henderson CE, Maragakis NJ. Gene profiling of human induced pluripotent stem cell-derived astrocyte progenitors following spinal cord engraftment. Stem Cells Transl Med. 2014 May;3(5):575-85. PubMed PMID: 24604284; PubMed Central PMCID: PMC4006486. 

5.Haidet-Phillips AM, Gross SK, Williams T, Tuteja A, Sherman A, Ko M, Jeong YH, Wong PC, Maragakis NJ. Altered astrocytic expression of TDP-43 does not influence motor neuron survival. Exp Neurol. 2013 Dec;250:250-9. PubMed PMID: 24120466.

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