Constructing the Future, Restoring the Past
Saturday, June 8 • 3:30pm - 5:30pm
Novel Biomarkers and Surrogate Endpoints Clarify Sickle Cell Disease Status and the Effects of Disease-Modifying Therapies

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The clinical pain and fatigue characteristic of sickle cell disease is difficult to objectively quantify.  Clinicians cannot depend on their physical exam, imaging studies, or laboratory data to validate claims of pain by their patients.  This challenge often creates physician and patient dissatisfaction, and makes it difficult to assess individual patient response to SCD modifying therapy in clinical trials. Biomarkers associated with cellular adhesion, inflammation, coagulation, anemia/hemolysis, cellular aggregates, microparticles and nitric oxide metabolism may help to objectively assess patient-reported outcomes (PROs) and response to disease modifying therapy.  There is an urgent need for validated biomarkers in sickle cell disease that facilitate open communication between the healthcare team, industry, and individuals with SCD.  We will highlight the current “state of the art” in the application of biomarkers and surrogate endpoints as applied to SCD in this educational session.
Session Objective
1.  Learn the principles of functional biomarkers and their applications to sickle cell disease.
2.  Learn how novel functional biomarkers can be applied to better understand the relationship between SCD biology, clinical manifestations, and patient experience.  
3.  Learn how novel biomarkers and surrogate endpoints can be incorporated into clinical trials of SCD-modifying agents.
4.  Understand how SCD biomarkers and surrogate endpoints are evaluated from a regulatory perspective.
Introductions, Overview of Definitions and Specific Biomarkers Currently Used in SCD Clinical Trials


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
Industry Perspective on Biomarkers and Surrogate Endpoints in SCD Clinical Trials

Kenneth R. Bridges, MD
Vice President of Medical Affairs
Global Blood Therapeutics
 Industry Perspective on Biomarkers and Surrogate Endpoints in Clinical Practice

Charles Stark, PharmD
Senior Vice President of Research and Development
Emmaus Life Sciences, Inc

avatar for Patrick Hines, MD PhD

Patrick Hines, MD PhD

Founder & Chief Executive Officer, Functional Fluidics
Patrick Hines, MD PhD is an Assistant Professor of Pediatric Critical Care Medicine and Associate in the Department of Physiology, Wayne State University School of Medicine., Children’s Hospital of Michigan. Previous business and technology consultant for Quantum Genomics, University... Read More →

avatar for Charles Stark, PharmD

Charles Stark, PharmD

Senior Vice President of Research and Development, Emmaus Life Sciences, Inc
avatar for Kenneth R. Bridges, MD

Kenneth R. Bridges, MD

Vice President, Medical Affairs, Global Blood Therapeutics
Dr. Kenneth R. Bridges received the MD degree from Harvard Medical School, and subsequently trained in internal medicine and hematology in Boston, at Massachusetts General and Brigham and Women’s Hospitals, respectively. Following medical subspecialty training, Dr. Bridges worked... Read More →

Saturday June 8, 2019 3:30pm - 5:30pm
Caribbean I Fort Lauderdale Marriott Harbor Beach

Attendees (31)