5th International RASopathies Symposium: When Development and Cancer Intersect
Renaissance Orlando, Florida ~ Friday, July 28 to Sunday, July 30, 2017
Chairs: Katherine A. Rauen, MD, PhD and Frank McCormick, PhD, FRS
Co-Organizers: RASopathiesNet
Lisa Schoyer, MFA, Lisa Schill, BS, Beth Stronach, PhD
AGENDA
[click HERE to download]
DAY 1 – FRIDAY Night 7/28/17 – Atrium A & B
8:00-10:00pm |
Dessert and Scientific Poster Session (Scientists and advocacy/family groups) |
DAY 2 – SATURDAY 7/29/17 – Crystal E
7:00-8:00 am |
Breakfast (Crystal D) |
9:00-5:00 pm |
Family Photo Shoot in and around meeting areas – Rick Guidotti |
8:00-10:00 am |
Session 1: What Defines a RASopathy? |
8:00-8:30 am |
Four Individuals/Caregivers: Perspectives from the Home Front |
8:30-9:00 am |
Frank McCormick, PhD, FRS: Molecular primer on the Ras signaling pathway |
9:00-9:30 am |
Katherine A. Rauen, MD, PhD: The RASopathies |
9:30-10:00 am |
Discussion (Clinical Diagnosis, Phenotype-Centered vs. Molecularly Defined) |
10:00-10:10 am |
Break |
10:10 am-12:00 pm |
Session 2: Syndromic and Sporadic Cancers of the Ras Pathway |
10:10-10:50 am |
KEYNOTE: Nancy Ratner, PhD: Preclinical studies to guide NF1 clinical trials |
10:50-11:10 am |
Corinne Linardic, MD: Pediatric sarcomas, rhabdomyosarcoma |
11:10-11:30 am |
Hélène Cavé, PharmD PhD: Myoproliferative neoplasms (JMML) in RASopathies |
11:30-11:50 am |
Brigitte Widemann, MD: Clinical trial updates, NF1 Plexiforms and MPNSTs |
11:50 am-12:00 pm |
Discussion |
12:00-1:00 pm |
Lunch and Learn: Rick Guidotti: Positive Exposure (Crystal D) |
1:00-4:30 pm |
Session 3: Human Development: Effects on Organ Systems |
1:00-1:40 pm |
KEYNOTE: Marco Tartaglia, PhD: RASopathy genetics, new mutations, congenital defects |
1:40-2:00 pm |
NERVOUS SYSTEM |
1:40-2:00 pm |
Erika Yeh, PhD (Weiss Lab): From skin cells to neurons |
2:00-2:20 pm |
Erik Ullian, PhD: How do astrocytes affect brain function in RASopathies? |
2:20-2:40 pm |
Giuseppe Zampino, MD: Penn MDBR 2015 Grantee – Pain in RASopathies: new investigative techniques and treatments |
2:40-2:50 pm |
Discussion |
2:50-3:00 pm |
Break |
3:00-3:40 pm |
CIRCULATORY SYSTEMS |
3:00-3:20 pm |
Bruce Gelb, MD: Human inducible pluripotent stem cells for the study of heart defects |
3:20-3:40 pm |
Max Itkin, MD: Imaging of the central lymphatic system in patients with Noonan syndrome using Dynamic Contrast Enhanced MR Lymphangiography |
3:40-4:00 pm |
GASTROINTESTINAL SYSTEM Cheng Sun, PhD (Kontaridis Lab): Penn MDBR 2014 Grantee – Using human inducible pluripotent stem cells to delineate the cause of gastrointestinal abnormalities in RASopathy disorders |
4:00-4:10 pm |
Discussion |
4:10-4:20 pm |
Break |
4:20-5:50 pm |
Session 4: Developmental Perspective: Modeling RASopathies in Animals and in Silico |
4:20-4:40 pm |
Stanislav Shvartsman, PhD: Quantitative biology of RASopathies |
4:40-5:00 pm |
Ethan Perlstein, PhD (Perlara): Rare disease drug discovery using whole animal disease models |
5:00-5:20 pm |
Edward Stites, MD, PhD: Computational analysis of pathological Ras mutants |
5:20-5:40 pm |
Annette Schenck, PhD: Habituation learning in Drosophila – a high-throughput platform to identify drugs that ameliorate cognitive and behavioural problems in Rasopathies |
5:40-5:50 pm |
Discussion |
5:50-6:30 pm |
Break |
6:30-8:30 pm |
Networking Dinner with Cash Bar |
DAY 3 – SUNDAY 7/30/17 – Crystal E
7:00-8:00 am |
Breakfast Meeting: How Do You Define a RASopathy? (Crystal D) |
8:00-9:30 am |
Session 5: Ras Pathway Mechanics |
8:00-8:20 am |
William Huang, PhD: Membrane signaling dynamics |
8:20-8:40 am |
John Albeck, PhD: Quantifying the cellular effects of Ras pathway mutations with live-cell imaging |
8:40-9:00 am |
Deborah Morrison, PhD: Divide and Conquer: Targeting Raf Regulatory Interactions |
9:00-9:20 am |
Marc Therrien, PhD: Allosteric control of RAF activation by dimerization |
9:20-9:30 am |
Discussion / Break |
9:30-11:00 am |
Session 6: Potential Therapeutics |
9:30-9:50 am |
Brage Andresen, PhD, FRCPath: Splice switching oligonucleotides (SSOs) for HRAS exon 2 skipping |
9:50-10:10 am |
Steven Fruchtman, MD (Oncovera Therapeutics): Rigosertib for JMML |
10:10-10:30 am |
Christopher Gibson, MD, PhD (Recursion Pharmaceuticals): Image-based high throughput screens for rare disease therapeutics |
10:30-10:50 am |
Philip Stork, MD: Penn MDBR 2016 Grantee – What can Ras-dependent cancers teach us about Rasopathies? |
10:50-11:00 am |
Discussion / Break |
11:00 am-12:30 pm |
Session 7: Next Generation: Junior Investigator Poster Session Abstracts and Closing Keynote Moderator: Bronwyn Kerr, MBBS Goal: Attract and highlight research on RASopathies from junior investigators |
11:00-11:30 am |
Junior Investigator Poster Finalists: Three 10-minute presentations, winner announced |
11:30-12:20 pm |
KEYNOTE: Frank McCormick, PhD, FRS: Targeting Ras and NF1-related malignancies |
12:20-12:30pm |
Discussion and Next Steps |
12:30-1:30 pm |
BREAKOUT SESSIONS
|
12:30-4:00 pm |
NF Network’s Post-Symposium Family Meeting (Coral B) |
2:30-4:30 pm |
RASopathy Advocacy Organizations’ Meeting on Approaching Data Collection and Sharing (Labrid B) |
2:30-6:00 pm |
Post-Symposium Events for CFC and NS families |
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