2021 MGFA Research Grant Recipients
The use of Survivin as a Diagnostic Marker for Myasthenia Gravis (Committed $55,000 per year for 2 years)
Dr. Linda Kusner M.D.
The George Washington University
Nine percent of patients with myasthenia gravis (MG) cannot have a clinical diagnosis confirmed by laboratory testing for detectable antibodies, designated seronegative MG (SNMG). We have found the expression of survivin in circulating lymphocytes to correlate with the diagnosis of acetylcholine receptor antibody-positive (AChR+) MG. We have also found survivin expressed in circulating lymphocytes from patients with muscle specific kinase antibody-positive (MuSK+) and rigorously defined SNMG patients, demonstrating the potential of survivin positivity as a diagnostic marker for MG. We propose to confirm positive survivin expression in circulating lymphocytes for the context of use as a diagnostic adjunct for MG.
Indoleamine-2, 3-dioxygenase 2 (IOD2) as a Novel Therapeutic Target for the Treatment of Myasthenia Gravis (Committed $55,000 per year for 2 years)
Dr. Laura Mandik-Nayak M.D.
Lankenau Institute for Medical Research
Myasthenia gravis (MG) is widely recognized as a B cell-mediated disease, with autoantibody production critical to its development and progression. While there has been intense interest in the development of therapies that deplete B cells or prevent B cell activation, these therapies are not effective in all patients and there is a continued need for new therapies. In this proposal, we will use a preclinical model of MG, together with a novel IDO2-targeting approach, to explore IDO2 inhibition as a novel therapeutic strategy to treat MG. In the short term, our studies will provide an initial step in the preclinical evaluation of IDO2 as a therapeutic target in the treatment of MG. If successful, the potential longterm impact of this project would move the concept of IDO2-directed therapy into development as a novel strategy to treat human MG.
- 2020 High Impact Pilot Project Grant Award: Dr. Kevin O’Connor, pHD, Yale University, "Identification of biomarkers that leverage mechanisms of autoantibody pathology in AChR MG" ($55,000 - One Year Award).
- 2020 High Impact Pilot Project Grant Award: Dr. Michael Hehir. M.D., University of Vermont, "Measuring adverse event burden in myasthenia gravis: Validation on adverse event unit" ($55,000 - One Year Award).
- 2020 Seronegative Grants: Dr. Jeffrey Guptill, M.D. – Duke University, Dr. Kevin O’Connor, pHD – "Yale University, Defining the clinical phenotype and immunopathology of seronegative MG" ($150,000 - 2 Year Award)
- 2019 High Impact Pilot Project Grant Award: Amanda C. Guidon, M.D., Massachusetts General Hospital, Neuromuscular Diagnostic Center, “Evaluation of automated techniques for decoding speech and movement abnormalities in myasthenia” $55,000
- 2019 High Impact Pilot Project Grant Award: Jeffrey T. Guptill, M.D., Duke University, “Metabolic Pathways of Pathogenic Th17 Cells in Myasthenia Gravis” $55,000
- 2019 High Impact Pilot Project Grant Award: Ricardo A. Maselli, M.D., University of California Davis, “CSF Delivery of AAV9-mediated gene therapy of congenital myasthenic syndrome due to CHAT mutations” $55,000
- 2020-2022 Research Contract: University of Alabama at Birmingham, "MG Patient Registry" $329,827
- 2018 High Impact Pilot Project Grant Award: Andrew Engel, M.D., Mayo Clinic, "Genetic Basis of Unsolved Congenital Myasthenic Syndromes and the Role of the AChR-CYS Loop Length in AChR Activation" $50,000
- 2017 High Impact Pilot Project Grant Award: David P. Richman, M.D., University of California, Davis, "Targeted Therapy of Myasthenia Gravis with Chimeric Autoantibody Receptor T Cells" $50,000
- 2016-2019 Clinician-Scientist Development Award: Post-doctoral fellowship to Michael Hehir, M.D., University of Vermont Medical Center, "Immunosuppressive Cost Unit: A Novel Method to Assess the Value and Cost of Immunosuppressant Side Effects" $160,000
- 2016-2018 Transformative Research Award for Myasthenia Gravis and Related Neuromuscular Junction Disorders: Jeffrey Guptill, M.D., Duke University, "Role of CD4 T cell Subsets as Drivers of MG Disease" $275,000
- 2016-2018 Research Opportunity Fund Award: Duke University, "PROMISE-MG Study meeting to expand research sites under Patient-Centered Outcomes Research Institute (PCORI) grant" $30,270
- 2016-2018 Research Contract: University of Alabama at Birmingham, "MG Patient Registry" $312,952
- 2016 Extension Research Opportunity Grant Award: Jeffrey Guptill, M.D., Duke University, "B10 Cells in MG/ Generate pilot polychromatic flow cytometry data on the role of B10 cells in a broad population of MG patients" $50,000
- 2016 Extension Research Opportunity Grant Award: Linda L. Kusner, Ph.D., George Washington University, "GWU/Anti-apoptotic Mechanisms in Persistence of Autoimmune Myasthenia Gravis" $50,000
- 2015 Continuation Research Opportunity Grant Award: Ricardo Maselli, M.D., University of California Davis, "Stem-cell treatment of congenital myasthenia associated with endplate acetylcholinesterase deficiency" $50,000
- 2015 Continuation Research Opportunity Grant Award: Ruksana Huda, Ph.D., University of Texas Medical Branch, "Novel cell specific therapy for autoimmune myasthenia" $50,000