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5:30 p.m. ~ Sunday, 13th January 2008
3:00 p.m. ~ Wednesday, 16th January 2008
Grand Hyatt Tampa Bay, Tampa, Florida, U.S.A.

Advanced Global Research

ALS Association Sponsored Scientific Workshop
Bringing together ALS researchers and non-ALS investigators, government, non profit, academia and industry to explore specific ALS-relevant topics and generate original
new research ideas

By invitation only

Reception Dinner
Sunday, 13th January 2008, 5:30 p.m. to 9:30 p.m.
Florida Aquarium, Tampa, Florida, U.S.A.

Arrival & Cocktails

Welcome Remarks
Dara Alexander, President, ALS Association Florida Chapter

ALS Association National Laureate
Noah benShea


Lucie Bruijn, Ph.D. Science Director and VP, ALS Association

Funding Opportunities for Translational Research at NINDS
Story Landis, Ph.D., NIH / NINDS

Keynote Speaker
Jeffrey Lichtman, Ph.D., Harvard University

Milton Safenowitz Post Doctoral Fellowship for ALS Research Awards


Scientific Workshop First Day
Monday, 14th January 2008, 8:00 a.m. to 6:00 p.m.
Grand Hyatt Tampa Bay, Tampa, Florida, U.S.A.

6:30-8:00    Breakfast
8:10-8:20    Introduction
Lucie Bruijn, ALS Association
8:20-10:30    Session 1: Overview of ALS
Chair: Christopher Henderson, Columbia University
8:30-9:10    Clinical picture of ALS
Kevin Boylan, Mayo Clinic
9:10-9:50    Genetics of familial and sporadic ALS
Teepu Siddique, Northwestern University
9:50-10:30    Unraveling the mechanisms involved in ALS
Don Cleveland, The Ludwig Institute for Cancer Research, UCSD
10:30-10:50    Break
10:50-1:00    Session 2: Drug discovery approaches for neurodegenerative diseases
Chair: Andrew Wood, Wyeth Research
11:00-11:30    Overview of drug discovery opportunities and challenges
John Kemp, Evotec
11:30-12:00    Screening approaches to identify compounds for neurodegenerative diseases
Christopher Lipinski, Melior Discovery Inc.
12:30-1:00    Discussion
1:00-2:00    Lunch
2:00-5:45    Session 3: Drug discovery approaches for ALS [continued on the second day]
Chair: Lucie Bruijn, ALS Association
2:00-2:30    Introduction to TREAT ALS - Translational Research Advancing Therapies for ALS
Lucie Bruijn, ALS Association
2:30-5:45    Session 3.1: Lessons learned from mutant SOD1: From target discovery to therapy
2:30-3:00    Development of a vaccine against mutant SOD1
Jean-Pierre Julien, Laval University
3:00-3:30    Protein aggregation and the development of ALS therapeutics
Donald Kirsch, Cambria Biosciences
3:30-4:00    Break
4:00-4:30    Small molecules against SOD1
Soumya Ray, Harvard University
4:30-5:00    Development of antisense therapy
Frank Bennett, ISIS
5:00-5:45    General discussion
Chair: Janice Robertson, University of Toronto
7:00      Cocktails and Dinner

Scientific Workshop Second Day
Tuesday, 15th January 2008, 8:00 a.m. to 5:00 p.m.
Grand Hyatt Tampa Bay, Tampa, Florida, U.S.A.

6:30-8:00    Breakfast
8:20-12:00    Session 3.2: Drug development in ALS: Identifying alternative targets
Chair: William Mobley, Stanford University
8:30-9:00    Axon dynamics
Erika Holzbaur, University of Pennsylvania
9:00-9:30    Role of inflammation in neurodegeneration
Tony Wyss-Coray, Stanford University
9:30-10:00    Mitochondria role in neurodegenerative diseases
Timothy Greenamyre, University of Pittsburgh
10:00-10:30    Break
10:30-11:00    The development of redox therapies for ALS
William Shrader, Edison
11:00-11:30    Development of non-target based assays for ALS: Phenotypic screen for ALS
Lee Rubin, Harvard Stem Cell Institute
11:30-12:00    Discussion
12:00-1:00    Lunch
1:00-2:00    Session 4: Challenges and Opportunities Learned from Other Neurodegenerative Diseases
Chair: Lorenzo Refolo, NIH / NINDS
1:00-1:30    Drug discovery in Huntington's Disease
Robert Pacifici, MRSSI/CHDI, Inc.
1:30-2:00    Drug discovery in Alzheimer's Disease
Ken Rhodes, Biogen-Idec.
2:00-2.30    Public - private partnerships: New opportunities for ALS research
Barbara Mittleman, NIH
2:30-2:50    Break
2:50-5:15    Session 5.0: Pre-Clinical and clinical challenges for ALS therapy development
(continued on third day)
       Session 5.1: Trial design, biomarkers and FDA regulatory issues
Chair: Al Sandrock, Biogen-Idec
3:00-3:30    Overview of clinical trials for ALS
Merit Cudkowicz, Harvard University & Massachusetts General Hospital
3:30-4:00    Use of biomarkers in ALS
Michael Strong, The University of Western Ontario
4:00-4:30    Issues and challenges in the design and execution of ALS clinical trials
Wilson Bryan, Biologics Consulting Group, Inc.
4:30-5:15    General discussion
6:00-7:30    Poster presentations
7:30      Dinner [arranged seating]

Scientific Workshop Third Day
Wednesday, 16th January 2008, 8:00 a.m. to 3:00 p.m.
Grand Hyatt Tampa Bay, Tampa, Florida, U.S.A.

6:30-6:30    Breakfast and early checkout
8:20-10:00    Session 5.2: Therapeutic delivery
Chair: Paul Sandberg, University of South Florida
8:30-9:00    Gene therapy approaches
Patrick Aebischer, Ecole Polytechnique Federale de Lausanne
9:00-9:30    Stem cell approaches
Clive Svendsen, University of Wisconsin
9:30-10:00    Discussion
10:00-10:30    Break
10:30-12:30    Session 5.3: ADME, preclinical toxicology and preparing for clinical development
Chair: Robert Pacifici, MRSSI/CHDI, Inc.
10:30-11:00    Translational opportunities at NIH - also with clinical trials
Lorenzo Refolo, NIH / NINDS
11:00-11:30    Screening of compounds in neurodegenerative models
Daniela Brunner, Psychogenics
11:30-12:00    ADME, preclinical toxicology
Janet Leeds, CHDI
12:00-12:30    Discussion
12:30-2:30    Working buffet lunch
12:30-2:30    Panel session: Future directions and opportunities for ALS drug discovery
Chair: Wim Robberecht, University of Leuven

Robi Blumenstein, CHDI
Christopher Henderson, Columbia University
Frank Longo, Stanford University
Andrew Wood, Wyeth
Martin Watterson, Northwestern University

Participant List

Steering Committee

William C. Mobley M.D., Ph.D.
D. Martin Watterson, Ph.D.
Frost White Ph.D.
Frank S. Walsh, Ph.D.
Alfred Sandrock, Ph.D.
Robert E. Pacifici, Ph.D.
Christopher E. Henderson, Ph.D.
Lorenzo M. Refolo Ph.D.


Jeff Lichtman
Kevin Boylan, M.D.
Teepu Siddique, MD
Don W. Cleveland
John A. Kemp, Ph.D.
Philip Tagari
Christopher A. Lipinski, Ph.D.
Alex Kiselyov
Jean-Pierre Julien
Richard Silverman
Soumya Ray
C. Frank Bennett Ph.D.
Janice Robertson
Erika L. F. Holzbaur, Ph.D.
Tony Wyss-Coray, Ph.D.
J Timothy Greenamyre, MD, Ph.D.
William D. Shrader, Ph.D.
Stephen Haggarty
Zuomei Li, Ph.D.
Lee Rubin
Merit Cudkowicz
Patrick Aebischer
Clive Svendsen, PhD
Daniela Brunner/Emer Leahy, Ph.D.
Janet Leeds
Barbara Mittleman
Jeffrey Rothstein
Frank M Longo, M.D, Ph.D.
Wim Robberecht


Raymond Bartus
Joseph Beckman
David Borchelt
Kyle Brown
Greg Cox
Brian Dickie
Denise Figlewicz
Jill Heemskerk
Donald Kirsch
Nigel Leigh
Jeffrey Macklis
Tom Maniatis
Richard Morimoto
Serge Przedborski
Mahendra Rao
Louis Reichardt
Raymond Roos
Paul Sandberg
Michael Strong
Zuoshang Xu

Last Modified May 21, 2012