Final Program

TUESDAY • MAY 17 239
• understand how PAH trials are designed and the basics of statistical
interpretation (e.g.-what is a hazard ratio?);
• understand the data emerging in PAH and how it applies to patients;
• outline the importance of various endpoints in PAH trials and why they are
important to patient care.
This session will focus on the wealth of clinical trials data in pulmonary
hypertension. Starting with a discussion of how such trials are designed and
then covering specific trials and endpoints will give the audience an overview
that they can apply to the care of patients with pulmonary arterial hypertension.
Chairing: R.N. Channick, MD, Boston, MA
V. McLaughlin, MD, Ann Arbor, MI
9:00 How Are Clinical Trials in PAH Designed:
The Basics
Speaker to be Announced
9:30 Comparing Endpoints in PAH Trials: Surrogate, Correlate, or
Something Else?
S.M. Kawut, MD, MS, Philadelphia, PA
9:50 PRO: Long-Term Morbidity Mortality Trials Are the Way to Go
in PAH
V. McLaughlin, MD, Ann Arbor, MI
10:05 CON: Event Driven Trials Are of Limited Value. Trials Need to
Be Designed to Determine if a Drug Actually Improves
Patients' Lives
V. Tapson, MD, West Hollywood, CA
10:20 PRO: Upfront Combination Therapy Should Now Be the
Standard of Care in PAH
M.J.C. Humbert, MD, PhD, Le Kremlin Bicetre, France
10:35 CON: Upfront Combination Therapy Has Not Been Proven to
Be Superior to Sequential Combination Therapy
R.N. Channick, MD, Boston, MA
10:50 Panel Discussion
R.N. Channick, MD, Boston, MA
C8
BASIC • TRANSLATIONAL
SCIENTIFIC SYMPOSIUM
CME Credits Available: 2.0
BREACHING BARRIERS: TARGETED DELIVERY OF
THERAPEUTICS TO THE LUNG
Assemblies on Respiratory Cell and Molecular Biology; Allergy, Immunology
and Inflammation; Clinical Problems; Critical Care; Pediatrics; Pulmonary
Circulation; Respiratory Structure and Function; Thoracic Oncology
9:00 a.m. - 11:00 a.m. MOSCONE CENTER
Room 3010/3012 (West Building, Level 3)
Target Audience
Pulmonary and critical care clinicians (adult, pediatric, neonatal) and lung
researchers.
Objectives
At the conclusion of this session, the participant will be able to:
• learn the latest findings about overcoming barriers to delivery of therapeutic
agents to the lungs;
• learn the advantages and limitations of endothelial vs epithelial targeting for a
variety of molecular entities in a range of lung diseases;
• apply knowledge regarding lung-specific delivery to overcome current limitations
in treatment of both rare and common lung diseases.
Many therapeutics which show promise in vitro are abandoned because of systemic
toxicity or inability to effectively penetrate lung tissue via airways or vasculature.
Emerging knowledge and technology is improving lung-specific delivery, eliminating
critical barriers in treatment of many lung diseases.
Chairing: J. Hagood, MD, La Jolla, CA
Z. Borok, MD, Los Angeles, CA
9:00 Caveolar Pumping for Lung Specific Delivery
J. Schnitzer, MD, La Jolla, CA
9:25 Mucus Penetrating Nanoparticles for Drug Delivery
J.S. Suk, PhD, Baltimore, MD
9:50 Using Nanocarriers to Target the Lung Endothelium
V.R. Muzykantov, MD, PhD, Philadelphia, PA
10:15 Mesenchymal Stem Cell Exosomes Target the Acutely
Injured Lung
M.A. Matthay, MD, San Francisco, CA
10:40 Panel Discussion
J. Hagood, MD, La Jolla, CA
C9
CLINICAL • TRANSLATIONAL
SCIENTIFIC SYMPOSIUM
CME Credits Available: 2.0
THE LINK BETWEEN CILIARY ASSEMBLY
DEFECTS, NEONATAL RESPIRATORY DISTRESS
AND BRONCHIECTASIS IN ADULTHOOD: A PRIMER
ON PRIMARY CILIARY DYSKINESIA
Assemblies on Pediatrics; Clinical Problems; Microbiology, Tuberculosis
and Pulmonary Infections; Respiratory Cell and Molecular Biology
9:00 a.m. - 11:00 a.m. MOSCONE CENTER
Room 3007/3009 (West Building, Level 3)
Target Audience
Pulmonologists, nurses, allied health professionals, clinical trainees,
translational scientists.
Objectives
At the conclusion of this session, the participant will be able to:
• acquire an understanding of how inherited defects in ciliary assembly
mechanisms lead to impaired ciliary structure and function;
• learn how to diagnose PCD with ciliary ultrastructural studies, genetic
studies and nasal nitric oxide measurement;
• understand the adverse health consequences of impaired ciliary function
from fetal development to adult life.
PCD is an inherited disease in which defects of the motile cilia lining the
respiratory tract cause impaired mucociliary clearance, resulting in progressive
TUESDAY MORNING
ATS 2016 • San Francisco