MSAB Research Agenda

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MSAB Research Agenda

• Henry J. Kaminski, MD
• Chair, MSAB

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The PastMGFA Sponsored Research

• MGFA Fellowships-Post Doctoral Fellowships
• Notable Fellows
• David Richman, MD-3 decades of research dedicated
  to MG-characterization of AChR structure,
  autoimmune response, therapeutic assessment,
  Co-organizer of International Conference
• Henry Kaminski, MD-2 decades of research
  dedicated to ocular muscle MG, therapeutic
  development, Co-organizer of International
  Conference

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The PastMGFA Sponsored Research

• MGFA Fellowships-Post Doctoral Fellowships
• Linda Kusner, PhD-6 years dedicated to MG
  research, ocular myasthenia, role of complement
  in MG pathology, MSAB member
• Erdem Tuzun, PhD-6 years dedicated to MG
  research, basic immunology of experimental
  myasthenia
• Numerous successful scientists not in MG, but
  with over 50 papers published in the area of MG

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The Past MGFA Sponsored Research

• The International meeting dedicated to MG
  Related Disorders

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The Past MGFA Sponsored Research

• Clinical Research Standards Taskforce (Chair
  Alfred Jaretzki)
• An internationally accepted set of standards
• Motivated clinical assessment research across the
  world
• Reassessment planned by MSAB, Chaired by Donald
  Sanders

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The Present MGFA Sponsored Research

• MGFA-AAN Fellowships
• 3 years of support for junior faculty to develop
  as scientists studying MG
• Jump starts career specific to MG
• Likely to stay in MG research
• Pipeline for the future

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The Present MGFA Sponsored Research

• MGFA supports a planning grant to evaluate
  Cellcept (mycophenolate mofetil) and clinical
  research standards
• MGFA has agreed to support a Genome Wide
  Association study of MG patients developed by
  Daniel Drachman
• MGFA support for the MGTX ancillary study
• MGTX is a NIH-sponsored trial that attempts to
  determine whether thymectomy is of added benefit
  to prednisone alone.
• MGFA supports a study of the thymus pathology,
  which otherwise would NOT have been done

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The Future-Research Agenda in Progress

• Recommendations 1 2 Support the future
  scientists through fellowships and junior faculty
• MGFA-AAN Research Fellowships
• Leverages MGFA resources
• Recommendations 3 4-Support novel opportunities
  and new approaches
• Opportunity Funding
• Genome-Wide Association Study

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The Future-Research Agenda in Progress

• Recommendation 5-Continue Student Summer Fellows
• Gets the MGFA name out across the country
• Recommendation 6-Yearly Scientific Session
• Gets the MGFA name out
• Opportunity for scientists to talk and establish
  collaborations

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The Future-Research Agenda in Progress

• Recommendation 7-International Meeting
• Established in the 1950s and regarded as the
  most important meeting in the field of Myasthenia
  Gravis Related Disorders. The Annals of the
  New York Academy of Sciences publication is a
  summary of all important developments.

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The Future-Research Agenda in Progress

• Recommendation 8-Summit to establish priorities
  for the Research Agenda
• MSAB will provide specific focus in next 6 months
• Recommendation 9
• Myasthenia Gravis Research Network
• Executive Committee agrees (note conflict of
  interest)
• Stimulus Challenge Grant submitted to NIH by Drs.
  Kaminski, Sanders, Cutter, Burns

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What is a MG Network?

• Group of collaborating investigators
• Submit uniform patient information
• Submit blood samples for a repository
• Value
• Ongoing monitoring of patient outcomes
• Preliminary data for planning clinical trials
• Biological samples for discovery

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What is the MG Network?
U of Texas, Southwestern Emory U U of
Rochester Kansas U Medical Center U of Illinois U
of Carolina, Chapel Hill U of California, Davis
University of Virginia
Data Management Coordinating Center University of
Alabama at Birmingham
Saint Louis University
Duke University
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Ongoing activities of MSAB

• Review and development of lay medical information
• Review of research grants
• Provide expert medical opinion to lay board
• Development of clinical research standards

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The FutureWhat is needed?

• Concentrated Effort by the MSAB and MGFA
• Financial Support of MGFA
• Financial Support from other sources
• NIH
• MDA
• Industry