13th Annual Angioma Alliance

CCM Scientific Meeting

Westin Georgetown, Washington, DC

October 26 & 27, 2017


Day 1 | Thursday October 26, 2017


8:50    Welcome & Opening Remarks        


Session I - Lesion Genesis


9:00    Uncovering molecular pathways involved in cerebral cavernous malformations by pharmacological suppression

            Cecile Otten - Potsdam University


9:15    The role of Polycomb group Proteins in CCM pathology

            Matteo Malinverno, Institute of Molecular Oncology, Milan


9:30    Inactivation of RhoA-kinase in Ccm3+/- mice suggest a role for the RhoA-kinase pathway in CCM3-lesion genesis and maturation

            Amy Peiper -  Duke University


9:45    Thrombospondin1 (TSP1) controls development of cerebral cavernous malformations

            Miguel Alejandro Lopez-Ramirez -  University of California, San Diego


10:00  Endothelial TLR4 and the gut microbiome drive cerebral cavernous malformations

            Alan Tang - University of Pennsylvania


10:15  Vascular Endothelial Growth Factor is required for CCM lesion initiation

            Kevin Whitehead - University of Utah and Angela Glading - University of Rochester


10:30  Discussion


10:45  Break


Session II - CCM Protein function


11:00  A conserved CCM complex promotes apoptosis non-autonomously by regulating zinc homeostasis in C. elegans

            Eric Chapman - University of Toronto


11:15  KRIT1 loss-of-function induces a chronic Nrf2-mediated adaptive homeostasis that sensitizes cells to oxidative stress: Implications for Cerebral Cavernous Malformation disease

            Cinzia Antognelli - University of Perugia


11:30  The role of Ccm2l in Cerebral Cavernous Malformations in neonatal mouse brain

            Jaesung Choi - Centenary Institute, Sydney


11:45  CCM3 deficiency increases levels of multiple tyrosine kinase receptors

            Juan Zalvide - Universidade de Santiago de Compostela


12:00  Discussion


12:30  LUNCH


Session III - Clinical Management and Patient Reported Outcomes


1:30    Review of a Large Registry of Pediatric Familial CCM

            Leslie Morrison - University of New Mexico


1:45    Minimally Invasive Resection of Rolandic Cavernomas in Children using a Novel Navigable Tubular Retractor System

            Erin Kiehna


2:00    Mental Health Outcomes in Pediatric Cavernoma

            Tiffany Campbell - Cincinnati Children's Hospital Medical Center


2:15    Quality of Life Measures in Familial Cerebral Cavernous Malformation Patients

            Jeff Nelson - University of California San Francisco


2:30    Discussion


Poster Session (3 – 4:30)


Pathologic/Radiologic Correlation and Findings in fCCM Lesions in Different Tissues

Blaine Hart – University of New Mexico


Role of lamotrigine in management of commonly encountered symptoms in patients with cerebral cavernous malformations

Asad Ikram -  University of New Mexico


Exploring the ccm-3 Network

Benjamin Lant – The Hospital for Sick Children


The Baca Family Historical Project: Paradigm-Shifting Approach to Patient Engagement

Connie Lee – Angioma Alliance


Up-regulation of NAPH oxidase-mediated redox signaling contributes to the loss of barrier function in KRIT1 deficient endothelium

Nicholas Nobiletti – University of Rochester


The Effect of Gut Microbiome on Chronic Models of CCM Lesion Formation

Heidy Pardo – Duke University


Development of the pectoral fin vasculature in zebrafish embryos

Scott Paulissen – NIH/NICHD


Characterizing the role of RHOA in regulating cranial vascular integrity

Laura Pillay – NIH/NICHD


ROCK2-selective inhibitors and their effects on key features of the biology of cerebral cavernous malformations

Jörg Ruschel – BioAxone Biosciences


A Novel Perivascular Cell Population in the Zebrafish Brain

Marina Venero Galanternik – NIH/NICHD


Novel functions of CCM1 delimit the relationship of PTB/PH domains

Jun Zhang – Texas Tech University Health Sciences Center


4:30    Break


Dinner (7-9 PM)

Nick's Riverside Grill, 3050 K St NW, Washington, DC




Day 2 | Friday, October 27, 2017


Session IV - Cell Biology  


9:00    The Cerebral cavernous malformation disease causing gene KRIT1 participates in epithelial barrier maintenance and regulation

            Le Shen - University of Chicago


9:15    Glycolytic metabolism and VEGFR3 signaling are required for lymphangiogenesis

            Joanne Chan - Hamilton University


9:30    Molecular regulation of vascular smooth muscle cell recruitment to arteries during development

            Amber Stratman – NIH/NICHD


9:45    Discussion


10:00  Keynote address

            Victoria Bautch - University of North Carolina at Chapel Hill


10:45  Break


11:00  Angioma Alliance Updates

            Connie Lee - Angioma Alliance


11:15  Panel Discussion – Pediatric CCM: Special Considerations for Treatment and Trials


Ed Smith, MD – Director Pediatric Cerebrovascular Neurosurgery, Boston Children’s


Barry Mangum, PharmD - CEO Paidion Research, Inc (Trial Design Considerations)

Ronald Farkas MD PhD - Vice President PAREXEL International (Regulatory Considerations)

Sudhakar Vadivelu, MD - Cincinnati Children’s Hospital and Medical Center (Clinical Management)


12:30  Lunch


Session V - Clinical Trial Readiness


1:30    Plasma Biomarkers of Inflammation and Angiogenesis Predict Cerebral Cavernous Malformation Symptomatic Hemorrhage or Lesional Growth

            Romuald Girard - University of Chicago


1:45    Developing a new drug for CCM: the path from bench to bedside

            Ken Rosen - BioAxone BioSciences


2:00    Recent Developments in Advancing Tempol as a Treatment for CCM

            Tim Considine - Recursion Pharmaceuticals


2:15    CASH (Cavernous Angiomas with Symptomatic Hemorrhage) Trial Readiness

            Sean Polster – University of Chicago


2:30    Discussion


2:45    2017 CCM Meeting Summary and Closing Remarks

            Issam Awad - University of Chicago


3:00    Close of Meeting