# Explore MG

> **NIH NIH U54** · GEORGE WASHINGTON UNIVERSITY · 2022 · $229,733

## Abstract

Myasthenia gravis (MG) treatment consists largely of toxic immunosuppressive regimens used
for years and utilized in a highly variable fashion among clinicians worldwide and with patients
complaining of a poor quality of life. Further complicating care is the growing appreciation over
the last few decades of clinical and pathophysiological heterogeneity. Presently, five distinct
subgroups of MG are generally accepted to exist: 1) early-onset acetylcholine receptor (AChR)
antibody (Ab) positive MG, which primarily affects women, 2) late-onset AChR Ab positive MG
with a disease bias towards men 3) paraneoplastic thymoma-associated MG, 4) muscle specific
kinase (MuSK) Ab positive, and 5) AChR/MuSK Ab negative MG. Only AChR Ab positive MG
has been well represented in clinical trials, in large part because of a lack of knowledge about
the natural history and treatment response in the other subgroups. There is a critical need to fill
this knowledge gap in order to allow for future clinical trials and improve patient care. Current
biomarkers available for use in the MG clinic do not predict response to immunosuppressive
therapies. Comprehensive immunologic profiles over the course of treatment must be performed
in large cohorts of MG patients. To address these deficiencies in research and clinical care, the
MGNet RDCRN proposes the Exploring Outcomes and Characteristics of Myasthenia Gravis
(EXPLORE-MG) study which will perform rigorous clinical phenotyping linked to standardized
biospecimen collection. We exploit the existing biorepository developed by the PI
(Neuromuscular IMmunology BiosampLE (NIMBLE) Network) created with the purpose of
biomarker discovery. We will integrate and expand the existing collaborative infrastructure with
a refined and extensive clinical phenotypic characterization developed by Dr. Richard Nowak at
Yale University. We propose Specific Aim 1: Characterize the clinical phenotype,
epidemiology, and diagnostic and management paradigms in the subgroups of MG and
Specific Aim 2: Enhance an existing multi-center biospecimen collection process to support
MGNet clinical projects 2 and 3, MGNet pilot studies program, and the greater MG research
community.

## Key facts

- **NIH application ID:** 10437796
- **Project number:** 5U54NS115054-04
- **Recipient organization:** GEORGE WASHINGTON UNIVERSITY
- **Principal Investigator:** Jeffrey T Guptill
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $229,733
- **Award type:** 5
- **Project period:** 2019-09-01 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10437796

## Citation

> US National Institutes of Health, RePORTER application 10437796, Explore MG (5U54NS115054-04). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10437796. Licensed CC0.

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