ACCELERATING ALS RESEARCH: TRANSLATING BASIC DISCOVERIES INTO THERAPIES FOR ALS

ACCELERATING ALS RESEARCH:
TRANSLATING BASIC DISCOVERIES INTO THERAPIES FOR ALS

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

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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

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

5:30
Arrival & Cocktails

6:00
Welcome Remarks
Dara Alexander, President, ALS Association Florida Chapter

6:15
ALS Association National Laureate
Noah benShea

6:30
Dinner

7:45
Lucie Bruijn, Ph.D. Science Director and VP, ALS Association

8:00
Funding Opportunities for Translational Research at NINDS
Story Landis, Ph.D., NIH / NINDS

8:20
Keynote Speaker
Jeffrey Lichtman, Ph.D., Harvard University

9:00
Milton Safenowitz Post Doctoral Fellowship for ALS Research Awards

9:15
Desert

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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

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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]

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

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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.

Speakers

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

Invitees

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