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lgelb

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Note: Summit in Central Time zone (New Orleans).


Day 1 https://www3.gotomeeting.com/register/740867726


Day 2 https://www3.gotomeeting.com/register/505585422



GLEASON SUMMIT

Round Table 1
Disease Mechanisms for ALS
Goal of Session: Highlight potential mechanisms in ALS, are they early, late, common
among other neurodegenerative diseases, common vs diverging pathways for ALS; Motor
neuron vs cortical neuron in light of FTD/ALS commonality; mindful of how to compare
disease mechanisms from model systems and in actual disease in humans, discussion of
biomarkers, prioritization of mechanisms.
• David Borchelt, Ph.D., University of Florida
• Nicolas Bazan, M.D., Ph.D., Louisiana State University Health Sciences Center
• Neil Cashman, M.D., University of British Columbia
• Piera Pasinelli, Ph.D., Thomas Jefferson University
• Wim Roberecht, M.D., Ph.D., University Hospitals Leuven, (Panel Co‐Chair)
• Jeffrey Rothstein, M.D., Ph.D., Johns Hopkins University, (Panel Co‐Chair)
• Paul Taylor, M.D., Ph.D., St. Jude Children’s Research Hospital

Round Table 2
Target Validation and Drug Development
Goal of Session‐prioritization of targets, how best to validate, what are the industry
standards, how can industry work with academia to move more quickly? What is Target
ALS doing? Mandatory need for pharmacodynamic markers; reliance on preclinical animal
models vs. human tissue? What are the gaps that other funding initiatives can support?
• Mike Alijanian, Ph.D., Bristol‐Myers Squibb, (Panel Co‐Chair)
• Christine Bulawa, Ph.D., Pfizer
• Jonathan Glass, M.D., Emory University
• Orest Hurko, M.D., EMD Serono
• Rita Sattler, Ph.D., Johns Hopkins University
• Frank Walsh, M.D., Ossianix Inc., (Panel Co‐Chair)

Round Table 3
Animal Models
Goal of Session; limitations, what have we learned in human translation? Safety
readouts? Value, ease of distributing models to community, have non vertebrate model
informed? Centralization of models, standardization, how to use animal models in
conjunction with human tissue and others models to validate targets, approaches to
making better animal models, talen technologies, other.
• Lucie Bruijn, Ph.D., The ALS Association
• David Borcelt, Ph.D., University of Florida, (Panel Co‐Chair)
• Terry Heiman Patterson, M.D., Drexel University
• Cat Lutz, Ph.D., The Jackson Laboratory, (Panel Co‐Chair)
• Udai Pandey, Ph.D., Louisiana State University
• Phil Wong, Ph.D., Johns Hopkins University

Round Table 4
Clinical Trials, Design, Challenges, Shortfalls
Some of the themes of recent meeting appropriate NeuroNext centralized IRB, phase II
trials are they adequate to tell us whether to go to phase II, how do we ensure
pharmacodynamics markers are available, are there mandatory prerequisites for
preclinical data before human trials? Stratification.
• Merit Cudkowicz, M.D., MSc, Massachusetts General Hospital, (Panel Co‐Chair)
• Eva Feldman, M.D., Ph.D., University of Michigan Health System
• Orest Hurko, M.D., EMD Serono
• Robert Miller, M.D., California Pacific Medical Center
• Mercedes Paredes, M.D., Ph.D., University of California San Francisco
• Jess Rabourn, Managing Director, The ALS Emergency Treatment Fund
• Jeffrey Rosenfeld, M.D., Ph.D., FAAN, University of San Francisco, Fresno, (Panel Co‐
Chair)
• Jeffrey Rothstein, M.D., Ph.D., Johns Hopkins University, (Panel Co‐Chair)

Round Table 5
Consortium Funding and Project Opportunities
Highlight current opportunities that would align well with a focused initiative and
directed funding.
• Lucie Bruijn, Ph.D., The ALS Association, (Co‐Chair)
• Amelie Gubitz, Ph.D., NINDS, NIH
• Jane Larkindale, Ph.D., MDA, (Co‐Chair)
• Melanie Leitner, Ph.D., Prize4Life
• Piera Pasinelli, Ph.D, Thomas Jefferson University
• Alvin Shih, M.D., Pfizer

Round Table 6
IPS cells, Disease Mechanism and Drug Discovery
Value as model system, biomarker development, what are industry perspectives,
phenotypes, standardization, resource sharing, specific patient lines of interest, C9,
TDP43 access for others globally, evidence for cross validation of an animal models vs.
human cell lines?
• Christine Bulawa, Ph.D., Pfizer
• Steve Finkbeiner, M.D., Ph.D., University of California San Francisco
• Nick Maragakis, M.D. Johns Hopkins University, (Panel Co‐Chair)
• Lee Rubin, Ph.D., Harvard Stem Cell Institute
• Clive Svendsen, Ph.D., Cedars‐Sinai, (Panel Co‐Chair)


Round Table 7
Patient Technologies
We need to find better approach/tools to comprehensively integrate patient and their
environment via technology‐ voice assistance, voice banking, assistive motor
technologies, integrated home technologies; brain interfaces
• Gregory Hager, Ph.D., Johns Hopkins University
• Bill Smart, Ph.D., Oregon State University

Round Table 8
Biomarkers
Current status, gaps, how best to centralize, partnerships with industry academia, what
are the current top biomarkers, confidence in each, what is required for further
validation, what studies need to be done that significant dollars could impact, pre‐FALS
studies, types of biomarkers, imaging tools for preclinical and clinical approaches,
pharmacodynamics biomarkers, current initiatives, what would move this field forward
faster, what is the industry doing?
• Mike Alijanian, Ph.D., Bristol‐Myers Squibb, (Panel Co‐Chair)
• Robert Bowser, Ph.D., Barrow Neurological Institute, (Panel Co‐Chair)
• Jonathan Glass, M.D., Emory University, (Panel Co‐Chair)
• Mike McGrath, M.D., Ph.D., University of California San Francisco
 

rknt50a

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bump to top... Timely info.
 

rknt50a

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Roundtables 6,7,8 today. If you are at all interested in ALS research, I encourage you to fire up the webstream at

Team Gleason | Steve Gleason | New Orleans Saints | summit

Yesterday there were hundreds watching/listening online. There was also a lively group chatting and asking questions.

Please spread the word. This is an unprecedented opportunity and we got such late notice that I'm guessing a lot of people aren't aware of it.

p.s. Although we hear a lot of the same ol same ol, the format doesn't let anyone put us into a coma with a long presentation. The comments are short, and they have a facilitator, Walter O'Brien, in the wings who issues some very interesting challenges to the scientists.
 
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