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SYMPOSIUM THEME
Hemoglobin S - Making an Evolutionary Perfected Process, Imperfect 

Journal of Sickle Cell Disease and Hemoglobinoopathies
SYMPOSIUM ABSTRACTS

Meeting Location:

Greater Ft. Lauderdale Convention Center
1950 Eisenhower Blvd, Fort Lauderdale, Florida, USA  33316
April 28 - 30, 2017 
Website:  fscdr.org
Email:  symposium@fscdr.org
Is this a CME Medical Meeting?  Yes
 
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Saturday, April 29 • 4:00pm - 6:00pm
Biomarkers and Surrogate Endpoint: Can Novel Biomarkers Clarify Sickle Cell Disease Status and Effects of Disease-Modifying Therapies? Current Challenges - Futures Opportunities

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Session Objective

  1. Learn the principles of important emerging functional biomarkers and their applications to sickle cell disease.
  2. Learn how novel functional biomarkers, electronic patient reported outcome platforms, and actigraphy can be applied to better understand the relationship between cellular-level biology of SCD, clinical manifestations, and patient experience.  
  3. Learn the pitfalls of incorporating novel biomarkers and surrogate endpoints into clinical trials of SCD modifying agents.
  4. Understand the perspective of regulatory agencies in evaluating biomarkers and surrogate endpoints. 

Introduction
4:00pm – 4:05pm
Patrick Hines, MD/PhD
Associate Professor of Pediatric Critical Care Medicine
Children’s Hospital of Michigan
Research Associate, Department of Physiology, WSU School of Medicine
Functional Fluidics, Founder and CEO

“ELIPSIS: a natural history study to evaluate longitudinal changes in ePRO, actigraphy, and biomarkers that describe the patient-centered VOC experience in SCD”

David Beidler, PhD
Director, Translational Sciences, Rare Diseases Research Unit
Pfizer

“Clinical application of standardized microfluidic flow-based assays to assess adhesive properties of sickle blood.” 

Patrick Hines, MD/PhD
Associate Professor of Pediatric Critical Care Medicine
Children’s Hospital of Michigan
Research Associate, Department of Physiology, WSU School of Medicine
Functional Fluidics, Founder and CEO

“Novel assays of platelet and WBC function to assess SCD acuity: Role of Platelet monocyte aggregates and platelet neutrophil aggregates”

Larry Frelinger, PhD
Assistant Professor of Pediatrics, Harvard Medical School
Associate Director, Center for Platelet Research Studies
Research Associate in Medicine, Division of Hematology/Oncology
Boston Children's Hospital/Dana-Farber Cancer Institute

“Coagulation markers and soluble plasma markers in SCD”

Debra Pittman, PhD
Research Fellow
Hematology Lead
Pfizer Rare Disease Research Unit

“Implementation of novel surrogate endpoints in clinical studies of SCD Modifying Therapy”

Michael Callaghan, MD
Associate Professor of Pediatric Hematology/Oncology
WSU School of Medicine
Children’s Hospital of Michigan

“Biomarkers as Surrogate Endpoints in Clinical Studies of SCD Modifying Therapy:  The Global Blood Experience”

Josh Lerner, MD
Chief Medical Officer
Global Blood Therapeutics

Panel Question and Answer Session


Moderators
avatar for Patrick Hines, M.D., Ph.D.

Patrick Hines, M.D., Ph.D.

Founder, Functional Fluidics, LLC
Dr. Patrick Charles Hines is an associate professor of pediatric critical care medicine at the Children’s Hospital of Michigan, associate in the Department of Physiology at Wayne State University School of Medicine, and founder of Functional Fluidics. Dr. Hines has spent over 15 years investigating the mechanisms regulating microvascular blood flow in sickle cell disease, including sickle erythrocyte adhesion, leukocyte rolling, and platelet... Read More →

Speakers
avatar for Michael U. Callaghan, M.D.

Michael U. Callaghan, M.D.

Director, Children’s Hospital of Michigan Sickle Cell Center
Assistant Professor of Pediatric Hematology                                   | Children’s Hospital of Michigan                                                          | Wayne State University of Medicine... Read More →
avatar for Josh Lehrer, M.D.

Josh Lehrer, M.D.

Vice President, Clinical Development, Global Blood Therapeutics
Dr. Lehrer is a cardiologist, physician scientist and biotechnology executive. He is currently Vice President, Clinical Development at Global Blood Therapeutics, where he has overseen the clinical development program strategy and execution for GBT440, bringing this molecule through early stage studies into a pivotal Phase 3 program in Sickle Cell Disease. Prior to GBT, Dr. Lehrer led clinical development programs through proof of concept at Genentech in multiple indications and held key roles in business development and overseeing cardiovascular safety. Dr. Lehrer has served as adjunct faculty in the Division of Cardiology at Stanford School of... Read More →
avatar for Larry Frelinger, Ph.D.

Larry Frelinger, Ph.D.

Staff Scientist, Boston Children’s Hospital
Dr. Andrew L. “Larry” Frelinger III is Assistant Professor of Pediatrics at Harvard Medical School, Associate Director of the Center for Platelet Research Studies, Staff Scientist at Boston Children’s Hospital, and has a joint appointment at the Dana-Farber Cancer Institute. Dr. Frelinger is also an editor of the journal Platelets and co-chair of the International Society on Thrombosis and Haemostasis Scientific and Standardization Committee subcommittee on Platelet Physiology. Dr... Read More →
avatar for David Beidler, PhD

David Beidler, PhD

Director, Translational Sciences, Pfizer Global Research and Development - Rare Diseases Resarch Unit
As the Translational Sciences Lead for the phosphodiesterase type 9 inhibitor project, David contributed to the development, implementation, and analysis of pharmacodynamic biomarkers in the recent phase 1b study in stable sickle cell disease subjects.  David has over 17 years of pharmaceutical industry experience with biomarker/translational pharmacology strategy and clinical operations that span preclinical animal models, natural history/methodology studies, and early (Phase 1/2) clinical... Read More →
avatar for Debra Pittman, PhD

Debra Pittman, PhD

Hematology Lead Research Fellow, Rare Disease Research Unit Pfizer
Debra is currently a Research Fellow and Hematology Lead in the Rare Disease Research Unit at Pfizer. Her early training was in molecular biology, but throughout her career she has worked on models of disease and the translation of nonclinical studies into early clinical... Read More →


Saturday April 29, 2017 4:00pm - 6:00pm
Floridian Ballroom A & B 3rd Floor Fort Lauderdale Convention Center