2022 MGFA Research Grant Recipients
Clinician and scientist investigators are engaged in groundbreaking myasthenia research all over the world. The MGFA funds promising research studies and clinical trials to discover potential new treatments and methods of living a better quality of life with MG. Here are some of the latest research funding grant recipients.
Jackie McSpadden Post-Doctoral Fellowship Award
The MGFA Jackie McSpadden Post-Doctoral Fellowship Award was established in 2022 to support a postdoctoral investigator conducting translational research related to myasthenia gravis (MG). The fellowship grant is offered to promising recipients of MD, PhD or MD/PhD degrees when it appears that the program of training to be supported by the grant will enhance the likelihood that the trainee will perform meaningful and independent research relevant to MG in the future and obtain a suitable position that will enable them to do so. This award is named for Jackie McSpadden as a posthumous memorial to her fighting spirit in the face of her myasthenia gravis diagnosis.
Measuring AChR autoantibody effector functions in myasthenia gravis patients
Dr. Fatemeh Khani
Yale University School of Medicine
The MGFA has officially announced its inaugural award recipient. We congratulate Fatemeh Khani, PhD, a Postdoctoral Associate in the Departments of Neurology and Immunobiology at Yale University School of Medicine.
Dr. Khani’s proposed research project “Measuring AChR autoantibody effector functions in myasthenia gravis patients” as part of her postdoctoral fellowship will be funded specifically to support her salary over a period of 3 years ($75,000/year) and will officially begin on January 1, 2023. Her fellowship training will be conducted in the Laboratory of Dr. Kevin O’Connor at Yale University.
Research Summary: The project investigation is focused on providing the framework for the development of MG biomarkers that can directly help patients by predicting treatment efficacy and disease progression. Dr. Khani seeks to understand immune mechanisms underlying MG that are anticipated to more precisely define this heterogeneous disease. These collective studies will provide a set of well-characterized biomarkers which will serve as tools for the community to more accurately model AChR in vitro. In addition, the work will provide a framework for understanding the association between autoantibody binding properties and effector functions in MG and identify candidate biomarkers that may proactively predict response to therapeutic complement treatment and avoid severe side effects from unnecessary interventions.
In addition to gaining a deeper understanding of MG immunopathology, the work will generate forward looking deliverables that are relevant to MG patient care in terms of prognosis and personalized/tailored therapies. This project’s proposed outcome may provide a deeper understanding of the mechanisms underlying the production of autoantibodies – a highly important determination for both the patient and clinician.
Fatemeh Khani Habibabadi, PhD (Postdoctoral Associate, Yale University) Bio: Dr. Khani completed her bachelor’s degree in cell and molecular biology at Isfahan University, followed by her master’s and PhD degrees in molecular genetics with a special focus on the molecular approach to studying multiple sclerosis at Tarbiat Modares University, Tehran. Based on these academic experiences, she is trained in laboratory-based human immunology and has extensive experience with the approaches that will be used in this work.
Inaugural Nancy Law Impact Award - 2022
The Nancy Law Impact Award is an MG research funding opportunity named after former MGFA Board Chair and CEO, Nancy Law, who was a caring, close friend to so many people across the myasthenia community. The proposals for this grant award are focused on innovative patient outcome measurements, optimization of clinical research approaches/practices, and the application of translational biomarkers that will aid in further refining the current treatment paradigm.
Preclinical models and biomarkers for predicting MuSK-CAART clinical outcomes ($300,000 over 3-year period)
Dr. Aimee Payne
University of Pennsylvania
Research Summary: MuSK MG is caused by MuSK autoantibodies that lead to life-threatening muscle weakness, so the ideal therapy would be to eliminate autoantibody-producing B-cells while preserving healthy B-cells. CART cells in the body are currently being re-programmed to eradicate B-cell cancers, prompting researchers to explore whether this precision medicine can be used for other diseases like myasthenia gravis. The project researchers are testing a novel autoantibody receptor T-cell therapy designed to re-program MG patient T-cells to selectively kill anti-MuSK B-cells that cause MuSK MG. The research is designed to test the working hypothesis in hopes of leading to a safe and lasting disease response and develop protocols for the detection and characterization of MuSK-CAART to validate novel biomarkers. Dr. Payne presented her ongoing work in this area during the 2022 AANEM MGFA Scientic Session in September.
MGFA High-Impact Pilot Project Award Recipient for 2022
The MGFA High-Impact Pilot Project Awards are pilot studies typically leading to new federal, pharmaceutical, or private foundation supported investigations. These grants are awarded annually. Congratulations to Dr. Lindsey De Lott from the University of Michigan!
Advancing patient-centered care and research for ocular myasthenia gravis: Validation of a novel patient reported outcome measure ($50,000 funded over 1-year)
Dr. Lindsey De Lott, M.D.
University of Michigan
Research Summary: The ocular symptoms of Myasthenia Gravis (MG) are disabling and affect quality of life - the impact of double vision and droopy eyelids can be profound. We need to fully understand the extent of how ocular symptoms impact daily function. Patient-reported outcome measures (PROMS) are valuable tools for measuring the aspects of MG, such as double vision, that matter most to patients while enhancing patient-physician communication and support. However, there are no PROMs focused on the impact of the ocular symptoms of MG or OMG nor sufficient scales to measure OMG.
Dr. De Lott aims to conduct a multicenter validation of the patient questionnaire as a standalone PROM for OMG so it can be deployed within future clinical research and support patient-centered care.
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:
- 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:
- 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:
- 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:
- 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:
- 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