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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
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Appendix C

Workshop Agenda

Neurodegeneration: Opportunities for Collaboration Across Disease-Specific Research and Development Communities—A Workshop

April 30-May 1, 2012

Pew DC Conference Center 901 E Street, NW, Washington, DC

Background: Neurodegenerative diseases are becoming increasingly prevalent in the United States due to the aging population. Implications of these diseases are grave, both for individual and family quality of life and for health care costs. Recent findings have revealed potential commonalities and parallelisms in genetic and cellular mechanisms across neurodegenerative diseases. Enhanced sharing of research findings and collaboration across research communities could potentially help advance basic scientific knowledge about each disease and about neurodegeneration and neurodegenerative diseases in general. Furthermore, enhanced basic scientific understanding could facilitate therapeutics development for neurodegenerative disorders, including therapeutics that may address more than one neurodegenerative disease. This workshop will explore commonalities across neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and frontotemporal dementia, and identify potential opportunities for collaboration across the respective research and development communities. Speakers and participants will be invited from academia; pharmaceutical and biotechnology industries; government agencies such as the National Institutes of Health, the National Science Foundation, and the Department of Veterans Affairs; and disease advocacy groups.

Meeting Objectives: The objectives of this workshop are to look across the neurodegenerative diseases—including Alzheimer’s disease, Parkinson’s disease, ALS, and frontotemporal dementia—and

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

 

•    Identify and discuss commonalities related to genetic and cellular mechanisms.

•    Identify areas of fundamental science needed to facilitate therapeutics development.

•    Explore areas of potential collaboration among the respective research communities and sponsors.

DAY ONE: April 30, 2012

8:00 a.m. Welcome and Opening Remarks

STORY LANDIS, Workshop Co-Chair

Director

National Institute of Neurological Disorders and Stroke

   
 

JOHN TROJANOWSKI, Workshop Co-Chair

Co-director, Center for Neurodegenerative Disease Research

University of Pennsylvania

   
8:10 a.m. U.S. Department of Veterans Affairs and Neurodegeneration Research: Current Efforts and Future Goals

JOEL KUPERSMITH

Chief Research and Development Officer

U.S. Department of Veterans Affairs

SESSION 1: OVERVIEW OF COMMON FEATURES ACROSS NEURODEGENERATIVE DISEASES

Session Objectives: The objectives of this session are to provide a genetic, clinical, and pathological framework to the notion that commonalities exist across neurodegenerative diseases. While this meeting focuses on discrete diagnostic entities, it is likely that this section may use examples from entities that cross these boundaries. Specifically, this session will

 

•    Provide an overview of the genetic complexity of different neurodegenerative diseases.

•    Discuss common and distinguishing features of the genetics of different neurodegenerative diseases.

•    Discuss the clinical heterogeneity of monogenic disorders.

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

•    Describe and discuss how pathology is likely to inform us about etiologic overlap between entities and provide illustrative examples of this overlap.

•    Discuss the rationale for looking across neurodegenerative diseases to advance scientific understanding and explore innovative approaches to therapeutics development.

 

8:20 a.m. Genetic Overlap and Complexity of Phenotypical Expression

ANDREW SINGLETON, Session Chair

Senior Investigator, Laboratory of Neurogenetics

National Institute on Aging

   
8:30 a.m. Pathological Overlap

DENNIS W. DICKSON

Professor of Laboratory Medicine and Pathology

Mayo Clinic

   
8:40 a.m. Translational Route Challenges: Is Combining Diseases Informative or a Distraction?

ADRIAN J. IVINSON

Director, Harvard NeuroDiscovery Center

Harvard Medical School

   
8:50 a.m. Discussion Among Speakers and Attendees

SESSION 2: PROTEIN AGGREGATION IN NEURODEGENERATIVE DISEASES

Session Objectives: The objectives of this session are to look at protein aggregation across the neurodegenerative diseases—including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, ALS, and frontotemporal dementia—and

 

•    Highlight commonalities related to protein aggregation across these diseases, for example, autophagy.

•    Discuss promising opportunities for collaboration among the respective research communities.

•    Identify areas of fundamental research about protein aggregation that would facilitate biomarker and therapeutics development.

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

•    Identify the next steps that research sponsors, investigators, and others should take to facilitate collaborative research and drug development in this area, including frameworks for partnerships and collaboration.

 

9:10 a.m. Overview of Status of the Field and Session Objectives

JOHN DUNLOP, Session Co-Chair

Vice President, Discovery

Neuroscience Innovative Medicine Unit

AstraZeneca

   
 

LUCIE BRUIJN, Session Co-Chair

Chief Scientist

ALS Association

   
9:20 a.m. Proteostasis Challenges in Neurodegenerative Diseases

RICK MORIMOTO

Professor of Molecular Biosciences

Northwestern University

   
9:30 a.m. Discussion Among Speakers and Attendees
   
9:45 a.m. The Selective Degradation of Misfolded Proteins and Protection Against Neurodegenerative Diseases

ALFRED GOLDBERG

Professor of Cell Biology

Harvard Medical School

   
9:55 a.m. Discussion
   
10:10 a.m. BREAK
   
10:25 a.m. Autophagy in Neurodegenerative Disease

ANA MARIA CUERVO

Professor, Department of Developmental and Molecular Biology

Albert Einstein College of Medicine

   
10:35 a.m. Discussion
   
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
10:50 a.m. Protein Aggregation in ALS and Huntington’s Disease

CLAUDIO HETZ

Professor, University of Chile

Adjunct Professor, Harvard School of Public Health

   
11:00 a.m. Discussion
   
11:15 a.m. Development of Assay Systems in Observing Aggregates and Development of Small Molecules

STEVEN FINKBEINER

Director, Taube-Koret Center, Gladstone Institute for Neurodegenerative Disease Professor, University of California, San Francisco

   
11:25 a.m. Discussion
   
11:40 a.m. Drug Discovery Efforts

WARREN HIRST

Associate Research Fellow, Neurodegeneration & Neurologic Diseases

Pfizer

   
11:50 a.m. Discussion
   
12:30 p.m. LUNCH

SESSION 3: MITOCHONDRIAL PATHOLOGY AND NEURODEGENERATIVE DISEASE

Session Objectives: The objectives of this session are to look at mitochondrial pathobiology across the neurodegenerative diseases—including Alzheimer’s disease and other dementias, Parkinson’s disease, and ALS—and to

 

•    Highlight differences and commonalities related to mitochondrial dysfunction and pathology across the diseases.

•    Discuss opportunities for the development of mitochondriarelated biomarkers and therapeutic interventions.

•    Identify next steps that research sponsors, investigators, and others should take to facilitate collaborative research and drug development in this area, including frameworks for partnerships and collaboration.

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

 

1:30 p.m. Overview of Status of the Field and Session Objectives

LENNART MUCKE, Session Chair

Director and Senior Investigator, Gladstone Institute of Neurological Disease

Professor of Neurology and Neuroscience

University of California, San Francisco

   
1:40 p.m. Systems Biology and Disease

VAMSI K. MOOTHA

Professor

Department of Systems Biology, Harvard Medical School

Department of Medicine, Massachusetts General Hospital

   
1:50 p.m. Discussion Among Speakers and Attendees
   
2:05 p.m. Neuronal Cell Death in Human Neurological Disorders and Their Animal/Cell Models

LEE MARTIN

Professor of Pathology, Neuroscience

Johns Hopkins University

   
2:15 p.m. Discussion
   
2:30 p.m. Parkinson’s Disease

RICHARD J. YOULE

Senior Investigator

National Institute of Neurological Disorders and Stroke

   
2:40 p.m. Discussion
   
2:55 p.m. BREAK
   
3:10 p.m. ALS and Huntington’s Disease

NEIL KOWALL

Professor of Neurology and Pathology, Boston University

Chief of Neurology, VA Boston Healthcare System

   
3:20 p.m. Discussion
   
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
3:35 p.m. Alzheimer’s Disease

DOUGLAS C. WALLACE

Director, Center for Mitochondrial and Epigenomic Medicine

Michael and Charles Barnett Chair of Pediatric Mitochondrial Medicine and Metabolic Disease

The Children’s Hospital of Philadelphia

   
3:45 p.m. Discussion
   
4:45 p.m. Wrap-Up: Highlights and Key Themes of Day One

STORY LANDIS, Workshop Co-Chair

JOHN TROJANOWSKI, Workshop Co-Chair

   
5:00 p.m. ADJOURN DAY ONE

DAY TWO: May 1, 2012

8:00 a.m. Welcome and Objectives of Day Two

STORY LANDIS, Workshop Co-Chair

Director

National Institute of Neurological Disorders and Stroke

   
 

JOHN TROJANOWSKI, Workshop Co-Chair

Co-director, Center for Neurodegenerative Disease Research

University of Pennsylvania

SESSION 4: NEURODEGENERATIVE DISEASE TRANSMISSION AND IMMUNE THERAPY

Session Objectives: The objectives of this session are to

 

•    Provide an overview of the latest concepts on transmission of neurodegenerative diseases, including evidence that suggests that disease progression may occur through the cell-to-cell spread of pathological disease proteins.

•    Explore how targeting transmissible species of α -Synuclein as well as tau and Abeta using immune therapy may be used to treat Parkinson’s disease and Alzheimer’s disease, respectively.

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

•    Identify the next steps that research sponsors, investigators, and others should take to facilitate collaborative research and drug development in this area, including frameworks for partnerships and collaboration.

 

8:15 a.m. Overview of Status of the Field and Session Objectives

JOHN TROJANOWKSI, Session Chair

Co-director, Center for Neurodegenerative Disease Research

University of Pennsylvania

   
8:25 a.m. Transmission of Prions and Alzheimer’s Disease Abeta Amyloid

CLAUDIO SOTO

Professor of Neurology

Director, Center for Alzheimer’s Disease and Related Brain Disorders

The University of Texas Medical School at Houston

   
8:35 a.m. Discussion Among Speakers and Attendees
   
8:50 a.m. Transmission of Alzheimer’s Disease Abeta Amyloid

LARY C. WALKER

Research Professor of Neuroscience

Emory University

   
9:00 a.m. Discussion
   
9:15 a.m. Transmission of Alzheimer’s Disease Tau Amyloid

KAREN DUFF

Professor, Department of Pathology

Columbia University

   
9:25 a.m. Discussion
   
9:40 a.m. BREAK
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
   
10:00 a.m. Transmission of Parkinson’s Disease α-Synuclein Amyloid

VIRGINIA M.-Y. LEE

The John H. Ware 3rd Professor in Alzheimer’s Research

Department of Pathology and Laboratory Medicine

Director, Center for Neurodegenerative Disease Research

University of Pennsylvania School of Medicine

   
10:10 a.m. Discussion
   
10:25 a.m. α-Synuclein Immunization for Parkinson’s Disease

DORA GAMES

Head of Pharmacology

Neotope Biosciences

   
10:35 a.m. Discussion
   
10:50 a.m. Tau Immunization for Alzheimer’s Disease and Related Tauopathies

PETER DAVIES

Head

Litwin-Zucker Center for the Study of Alzheimer’s Disease and Memory Disorders

The Feinstein Institute for Medical Research

   
11:00 a.m. Discussion
   
11:45 a.m. LUNCH

SESSION 5: ERRORS IN RNA

Session Objectives: The objectives of this session are to

 

•    Discuss how errors in RNA-binding proteins are causes of neurodegenerative diseases, including ALS, frontotemporal dementia, and spinal muscular atrophy, as well as triplet nucleotide expansion as a risk factor in disease (e.g., ataxin and ALS).

Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×

•    Discuss disease mechanisms for diseases with toxic RNAs, including myotonic dystrophy and other triplet nucleotide repeats where there are toxic RNAs or aberrant translation of the expansions.

•    Explore potential biomarkers and therapies for RNA-binding protein errors in SMA, TDP-43, FUS, and C9orf72.

•    Discuss yeast models to identify therapeutics and the emerging roles of non-coding RNA networks in the pathogenesis of neurodegenerative diseases.

•    Identify the next steps that research sponsors, investigators, and others should take to facilitate collaborative research and drug development in this area, including frameworks for partnerships and collaboration.

 

12:45 p.m. Overview of Status of the Field and Session Objectives

DON CLEVELAND, Session Chair

Professor and Chair, Department of Cellular and Molecular Medicine

Head, Laboratory for Cell Biology

Ludwig Institute for Cancer Research

University of California, San Diego

   
12:55 p.m. Overview of RNA Gain-of-Function Mechanisms in Neurodegenerative Disease

LAURA RANUM

Professor of Molecular Genetics and Microbiology

University of Florida

   
1:05 p.m. Discussion
   
1:20 p.m. Overview of Therapies for RNA-Binding Protein Errors

FRANK RIGO

Assistant Director, Core Antisense Research

ISIS Pharmaceuticals, Inc.

   
1:30 p.m. Discussion
   
1:45 p.m. Yeast Models to Identify Therapeutics

GREGORY A. PETSKO

Gyula and Katica Tauber Professor of Biochemistry & Chemistry

Brandeis University

   
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
1:55 p.m. Discussion
   
2:10 p.m. The Emerging Roles of Non-Coding RNA Networks in the Pathogenesis of Neurodegenerative Diseases

MARK F. MEHLER

Alpern Professor of Neurology, Neuroscience and Psychiatry and Behavioral Sciences

University Chair, The Saul R. Korey Department of Neurology

Albert Einstein College of Medicine

   
2:20 p.m. Discussion
   
2:45 p.m. BREAK

SESSION 6: FUTURE DIRECTIONS AND NEXT STEPS

Session Objectives: A panel will synthesize and discuss key highlights from the workshop presentations and discussions, including

 

•    Identify key promising areas for future cross-disease research and collaboration.

•    Discuss opportunities for partnerships—public–private and across disease-specific communities—to advance neurodegeneration research and therapeutics development.

•    Discuss challenges to advancing research and therapeutics development for the neurodegenerative diseases and potential mechanisms to address these challenges.

 

3:00 p.m. Panel Discussion (Session Chairs from Previous Sessions):

STORY LANDIS, Session Co-Chair

Director

National Institute of Neurological Disorders and Stroke

   
 

JOHN TROJANOWSKI, Session Co-Chair

Co-director, Center for Neurodegenerative Disease Research

University of Pennsylvania

   
 

ANDREW SINGLETON

Senior Investigator, Laboratory of Neurogenetics

National Institute on Aging

   
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
 

JOHN DUNLOP

Vice President, Discovery

Neuroscience Innovative Medicine Unit

AstraZeneca

   
 

LUCIE BRUIJN

Chief Scientist

ALS Association

   
 

LENNART MUCKE

Director and Senior Investigator, Gladstone Institute of Neurological Disease

Professor of Neurology and Neuroscience

University of California, San Francisco

   
 

DON CLEVELAND

Professor and Chair, Department of Cellular and Molecular Medicine

Head, Laboratory for Cell Biology

Ludwig Institute for Cancer Research

University of California, San Diego

   
3:30 p.m. Discussion Among Speakers and Attendees
   
4:30 p.m. ADJOURN
Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Suggested Citation:"Appendix C: Workshop Agenda." Institute of Medicine. 2013. Neurodegeneration: Exploring Commonalities Across Diseases: Workshop Summary. Washington, DC: The National Academies Press. doi: 10.17226/18341.
×
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Neurodegeneration: Exploring Commonalities Across Diseases is the summary of a workshop hosted by the Institute of Medicine's (IOM's) Forum on Neuroscience and Nervous System Disorders in Spring 2012 to explore commonalities across neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD). Participants from academia; pharmaceutical and biotechnology industries; government agencies such as the National Institutes of Health and the U.S. Department of Veterans Affairs (VA); patient advocacy groups; and private foundations presented and identified potential opportunities for collaboration across the respective research and development communities. This report identifies and discusses commonalities related to genetic and cellular mechanisms, identifies areas of fundamental science needed to facilitate therapeutics development, and explores areas of potential collaboration among the respective research communities.

Neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease, ALS, and FTD, are becoming increasingly prevalent in the United States due to an aging population. Implications are grave for quality of life and health care costs. Research on neurodegenerative diseases has expanded greatly over the past four decades. Nevertheless, fundamental questions remain about the biology of these diseases, and further insights into the mechanisms of these diseases would help to inform the development of effective means to prevent and to efficiently treat them. Recent findings have revealed certain commonalities in genetic and cellular mechanisms across neurodegenerative diseases. These findings suggest that it might be valuable - at least in some cases - to change the traditional way of studying these diseases by no longer seeing each as an independent entity, but rather as clinical variants of common cellular and molecular biological defects. This approach could help enhance basic scientific understanding of neurodegenerative disease, and could help with the development of biomarkers and new therapeutics.

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