Myasthenia Gravis Foundation of America, Inc

Myasthenia Gravis Foundation of America, Inc


For the last 3 years, the MGFA has gone through a process of re-assessment of their research grant programs through a process of consultation with MGFA chapters, the MGFA Medical Scientific Advisory Board, other health advocacy groups, and leaders of federal research funding bodies, including the National Institutes of Health. This assessment has led to three specific initiatives:

  • Establishment of a set of MGFA Research Priorities (see below)
  • Restructuring of support for young investigators, which led to establishment of a joint venture with the AmericanAcademy of Neurology to support junior faculty research dedicated to MG
  • Establishment of the High Impact Pilot Projects for MG and Related Neuromuscular Junction Disorders


a)Etiology- what is the basic cause of the disease? Define genetic and environmental factors?

b)Detection/early diagnosis: although most patients can be diagnosed relatively early there are some who remain difficult to diagnose and some in whom there is uncertainty regarding the diagnosis. Can more sensitive and more specific methods of diagnosis be developed? Can we educate physicians and the public better to identify MG? Would earlier diagnosis lead to improved treatment?

c)Genetics of MG

1. Genome-wide association study (this is in development)

2. Other approaches

3. Proteomic evaluation

d)Enhance understanding and application of current treatment of MG : Application of drugs developed for use in other diseases and assessment of established therapy including mycophenolate mofetil (MMF), tacrolimus (FK 506), rituximab, anti-TNF agents (etanercept), and further study of a promising antisense oligonucleotid, Monarsen. Furthermore, are there particular combinations of treatments that will lead to the best responses?

Plasma Exchange and IVIg are remarkably effective treatment options but they are difficult to administer and they are extremely expensive. Is there a way to provide these treatments in an easier, safer, and less expensive format? Can we establish exactly why IVIg works in MG? Is immunoabsoption, a more specific approach than plasma exchange geared to only remove known-to-be pathogenic antibodies in the disease, a potential approach?

e)Thymus: how exactly does the thymus gland promote the production of acetylcholine receptor antibodies? Are we scientifically certain about the role of thymectomy in treating this disorder? The thymus is abnormal in MG. Does the type or degree of abnormality predict the clinical course of MG or optimal treatment?

f)New therapeutic targets need to be identified for MG, with the goal of improved treatment responses and fewer adverse events. Areas proposed for further study include complement inhibition and dendritic cells/antigen presenting cells.

g)New strategies for treatment include development of potential vaccine therapies:


Give AChR or synthetic AChR

Mucosal or subcutaneous

Effective in experimental MG

T-cell vaccines

Already in clinical trials in MS and rheumatoid arthritis

Effective in experimental MG

Induce antibodies that target the T cell receptor

Antigen-specific tolerance

Immune regulatory cells (T cells, B cells, Dendritic cells)

Regulatory antibodies

h)Quality of life aspects: develop better strategies to improve quality of life and develop rigorous research methods to study quality of life in MG.

i)Collateral effects of MG: sleep disorders, cognition, stress relationships, associated medical conditions, financial comparative effectiveness.

MGFA-AANF Fellowship

MGFA and AANF (American Academy of Neurology Foundation) are offering a joint three year Clinician Scientist Development fellowship. The fellowship is designed to train the next generation of clinical researchers.

High Impact Pilot Projects on Myasthenia Gravis and Related Neuromuscular Junction Disorders

The Myasthenia Gravis Foundation of America requests submission of proposals to support pilot studies that are highly focused and innovative with a clear plan that will lead to new federal, pharmaceutical, or private foundation supported investigations. The proposal should be within the scope of the research agenda of the Myasthenia Gravis Foundation, which may be found at The MGFA will fund highly meritorious projects with a maximum direct cost of $50,000 per year and a maximum 10% indirect cost rate. Consideration will be given to renew support on a yearly basis for a maximum of 2 years, but clear achievement of milestones must be demonstrated to justify renewal. Provide a detailed budget in the format used for NIH budgets. There are no specific budget restrictions but all aspects of the budget must be clearly justified. If PI salary support is requested, then a letter from a department chair or equivalent must accompany the application to assure protected research time is assured.

The application consists of a five page research section, which should include specific aims, background/preliminary data, innovation, and approach sections. There is no limit to the reference section. A separate single page section should describe how the funds will be used to develop future support. This section should be as specific as possible, and this future plans section is critical in the review process. A copy of a funding agency review, which supports the need to obtain preliminary data would be evidence of specific plan for future support. Such material may be included in an appendix.

Proposals will be reviewed by the MGFA research committee as well as ad hoc reviewers when required. The RFA is open to established and new investigators. The funding is restricted to principal investigators in academic institutions in Canada and the United States, although collaborators may reside outside North America.

Proposals should be submitted by email to Tor Holtan, () no later than January 15, 2011. A preliminary review to assure compliance with guidelines will be performed, followed by scientific review. Funding notification is expected by May 15, 2011 and funds provided by July 1, 2011. Three awards are expected to be given out.