Summit on Drug Discovery in TSC and Related Disorders
Sponsored by The Rothberg Institute of Childhood Diseases
Omni Shoreham Hotel, Washington, DC
Draft Conference Agenda
Wednesday, July 6, 2011
Noon - 8:30 pm
Registration (Blue Pre-function Room)
1:00- 2:15 PM
TSC Natural History Database Steering Committee Meeting
(Congressional B)
This is a strategic planning meeting with the Administrative Project Leader,
Co-Scientific Project Leaders, Working Group Leaders, and TSC database site
investigators and/or colleagues who are interested in being a committee
member in one of the working groups: epilepsy, genetics, neurocognition.
2:15 – 2:30 pm
Break
2:30 – 3:30 pm
The TSC Clinic Directors Meeting (Congressional B)
This meeting is for all TSC Clinic Directors and Nurse Coordinators.
Administrative and research coordinators are also welcome to attend. We
will review the TSC Clinic Guidelines; talk about current clinical trials and
have an open discussion about setting up a TSC clinical research network.
3:30-3:45 pm
Break
3:45–5:00 pm
TSC Natural History Database Project Meeting (Congressional B)
Attendees: TSC Database site principal investigators, database users,
interested researchers.
6:30 - 7:30 pm
Opening Reception (Blue Pre-function Room)
7:30 - 8:30 pm
Welcome and Introductions (Blue Room)
High Throughput Screening for TSC, LAM and Related Disorders
Chris Austin, M.D.
NHGRI, NIH, Bethesda, MD
Thursday, July 7, 2011
7:30 am - 5:00 pm
Registration (Blue Pre-function Room)
Session I: Drug Screening and Drug Discovery in TSC and Related Disorders
Session Chairs: David Schenkein, M.D. & Elizabeth Petri Henske, M.D.
(Blue Room)
7:30 – 8:45 am
Breakfast (Hampton Room)
9:00 – 9:10 am
Parent Speaker
9:10 – 9:40 am
Targeted Therapeutic Options and Drug Screens for TSC and Related
Disorders
Aristotelis Astrinidis, Ph.D.
Drexel University College of Medicine, Philadelphia, PA
9:40 – 10:10 am
Drug Screening for TSC: A National TS Alliance Resource
Summer Gibbs-Strauss
Beth Israel Deaconess Medical Center, Boston, MA
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10:10 – 10:30 am
TBD- Speaker selected from submitted abstracts
10:30 – 11:00 am
Break (Blue Pre-function Room)
11:00 – 11:20 am
TBD – Speaker selected from submitted abstracts
11:20 – 11:40 am
TBD – Speaker selected from submitted abstracts
11:40 am – Noon
TBD – Speaker selected from submitted abstracts
Noon – 1:30 pm
Lunch – Industry Panel Discussion
(Hampton Room)
Session II: Preclinical Studies in TSC and Related Neurological Diseases
Session Chairs: Petrus de Vries, M.D., Ph.D. & Vijaya Ramesh, Ph.D. (Blue Room)
1:30 – 2:00 pm
mTOR Inhibitor-based Modulation of Behavioral and Neurological Phenotypes
in Mouse Models of Tuberous Sclerosis
Dan Ehninger, Ph.D.
German Centre for Neurodegenerative Diseases (DZNE), Bonn, Germany
2:00 – 2:30 pm
Axonal Connectivity in Tuberous Sclerosis: Studies in Mouse Models and
Patients
Mustafa Sahin, M.D., Ph.D.
Children’s Hospital, Boston, Boston, MA
2:30 – 3:00 pm
Preclinical Studies for Developing Rational Therapies for Epilepsy in Tuberous
Sclerosis Complex
Michael Wong, M.D., Ph.D.
Washington University School of Medicine, St. Louis, MO
3:00 – 3:30 pm
Break (Blue Pre-function Room)
3:30 – 5:00 pm
Working Groups
A. Neurocognitive Outcomes in Clinical Trials for TSC and Related Disorders
(Congressional A)
Session Leaders: Petrus de Vries, M.D., Ph.D. & Mustafa Sahin, M.D.,
Ph.D.
B. Biomarkers for Translational and Clinical Studies in
TSC and Related Disorders (Congressional B)
Session Leaders: Frank McCormack, M.D. & Joel Moss, M.D., Ph.D.
5:00 – 7:00 pm
Poster Session and Reception (Blue Pre-function Room)
5:00 – 8:00 pm
Partnering Meetings (Location TBD)
Friday, July 8, 2011
7:30 – 8:45 am
Breakfast (Hampton Room)
8:50 – 9:00 am
Parent Speaker (Blue Room)
9:00 – 9:15 am
Report from Working Group A
9:15 – 9:30 am
Report from Working Group B
9:30 – 9:45 am
Discussion
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Session III: Preclinical Studies for Tumor Growth in TSC and Related Disorders
Session Chairs: David Kwiatkowski, M.D., Ph.D. & Julian Sampson, M.B.
9:45 – 10:15 am
Nutrients, mTOR Signaling and Cellular Metabolism
John Blenis, Ph.D.
Harvard Medical School, Boston, MA
10:15 – 10:35 am
TBD-Speaker Selected from Submitted Abstracts
10:35 – 11:00 am
Break (Blue Pre-function Room)
11:00 – 11:30 am
Progress in the Therapy of LAM: Bench to Bedside
Elizabeth Petri Henske, M.D.
Brigham & Women’s Hospital, Boston, MA
11:30 am – Noon
Speaker TBD
Noon – 1:30 pm
Lunch- NIH Panel- Process and Priorities of Drug Discovery at
NIH
(Hampton Room)
1:30 – 2:00 pm
Preclinical Studies in TSC Tumor and Brain Models (Blue Room)
David Kwiatkowski, M.D., Ph.D.
Brigham & Women’s Hospital, Boston, MA
2:00 – 2:20 pm
TBD – Speaker selected from submitted abstracts
2:20 – 2:40 pm
TBD – Speaker selected from submitted abstracts
2:40 – 3:00 pm
Break (Blue Pre-function Room)
3:00 – 4:30 pm
Working Groups
C. Drug Discovery for Treatment of Tumors in TSC and
Related Disorders (Congressional A)
Session Leader: John Blenis, Ph.D.
D. Neurological Outcomes in Clinical Trials for TSC and Related Disorders
(Congressional B)
Session Leaders: Michael Wong, M.D., Ph.D. & Peter Crino, M.D.,
Ph.D.
4:30 – 6:00 pm
Partnering Meetings (Location TBD)
6:00 – 8:30 pm
Summit Reception and Dinner Sponsored by Brian and Sandra O’Brien
Keynote Speaker: Jonathan Rothberg, Ph.D.
(Diplomat Room)
Saturday, July 9, 2011
7:30 – 8:45 am
Breakfast (Hampton Room)
8:50 – 9:00 am
Parent Speaker (Blue Room)
9:00 – 9:15 am
Report from Working Group C
9:15 – 9:30 am
Report from Working Group D
9:30 – 9:45 am
Discussion
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Session IV. Clinical Trials in TSC, LAM and Related Disorders
Session Chairs: Elizabeth Thiele, M.D., Ph.D. & Martina Bebin, M.D., MPA
9:45 – 10:15 am
Organizing Multicenter Clinical Trials: The NF Consortium
Bruce Korf, M.D., Ph.D.
University of Alabama at Birmingham, Birmingham, AL
10:15 – 10:45 am
Clinical Trials for Lymphangioleiomyomatosis & Biomarker Studies
Frank McCormack, M.D.
University of Cincinnati, Cincinnati, OH
10:45 – 11:15 am
Break (Blue Pre-function Room)
11:15 – 11:45 am
Clinical Trials in the UK for TSC and Related Disorders
Julian Sampson, MB
Cardiff University, Cardiff, Wales, UK
11:45 am – 12:15 pm
Clinical Trials in the USA for TSC and Related Disorders
David Neal Franz, M.D.
Cincinnati Children’s Hospital and Medical Center, Cincinnati, OH
12:15 – 12:30 PM
Discussion and Wrap-Up
12:30 pm
Summit Adjourns
2:00 – 6:00 pm
International Scientific Advisory Board Meeting (Dinner to follow at 7:00 pm)
Invitation Only
Attendees: TS Alliance International Scientific Advisory Board
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International TSC Research Conference Confirmed Speakers
Aristotelis Astrinidis, Ph.D., Drexel University College of Medicine, Philadelphia, PA
Chris Austin, M.D., NHGRI, NIH, Bethesda, MD
Martina Bebin, M.D., M.P.A, University of Alabama at Birmingham, Birmingham, AL
John Blenis, Ph.D., Harvard Medical School, Boston, MA
Peter Crino, M.D., Ph.D., University of Pennsylvania, Philadelphia, PA
Petrus de Vries, M.D., Ph.D., University of Cambridge, Cambridge, UK
Dan Ehninger, Ph.D., German Centre for Neurodegenerative Diseases, Bonn, Germany
David Neal Franz, M.D., Cincinnati Children’s Hospital, Cincinnati, OH
Summer Gibbs-Strauss, Ph.D, Beth Israel Deaconess Medical Center, Boston, MA
Elizabeth Petri Henske, M.D., Brigham & Women’s Hospital, Boston, MA
Bruce Korf, M.D., Ph.D., University of Alabama at Birmingham, Birmingham, AL
David Kwiatkowski, M.D., Ph.D., Brigham & Women’s Hospital, Boston, MA
Frank McCormack, M.D., University of Cincinnati School of Medicine, Cincinnati, OH
Joel Moss, M.D., Ph.D., NHLBI, NIH, Bethesda, MD
Vijaya Ramesh, Ph.D., Massachusetts General Hospital, Boston, MA
Jonathan Rothberg, Ph.D., Ion Torrent, Gilford, CT
Mustafa Sahin, M.D., Ph.D., Children’s Hospital, Boston, MA
Julian Sampson, M.D., Cardiff University, Cardiff, UK
David Schenkein, M.D., Agios Pharmaceuticals, Cambridge, MA
Elizabeth Thiele, M.D., Ph.D., Carol & James Herscot Center for TSC, Massachusetts General Hospital,
Boston, MA
Michael Wong, M.D., Washington University School of Medicine, St. Louis, MO
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International TSC Research Conference
Organizing Committee
Co-Chair:
Co-Chair:
Elizabeth Petri Henske, M.D., Brigham & Women’s Hospital, Boston, MA
Chair of the TS Alliance International Scientific Advisory Board
Peter Crino, M.D., Ph.D., University of Pennsylvania, Philadelphia, PA
Chair of the TS Alliance Professional Advisory Board
Organizing Committee Members:
E. Martina Bebin, M.D., MPA, University of Alabama, Birmingham, AL
Petrus de Vries, M.D., Ph.D., Cambridge University, Cambridge, UK
Brendan Manning, Ph.D., Harvard School of Public Health, Boston, MA
Vijaya Ramesh, Ph.D., Massachusetts General Hospital/Harvard, Boston, MA
Kari Luther Rosbeck, President & CEO, Tuberous Sclerosis Alliance, Silver Spring, MD
Mustafa Sahin, M.D., Ph.D., Children’s Hospital/Harvard, Boston, MA
Katie Smith, Science Coordinator, Tuberous Sclerosis Alliance, Silver Spring, MD
Elizabeth Thiele, M.D., Ph.D., Carol & James Herscot Center for TSC, Massachusetts General
Hospital, Boston, MA
Joseph Twist, PharmD., Tuberous Sclerosis Alliance, Silver Spring, M.D.
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