Glomerular Disease: Pathophysiology, Biomarkers, and Registries for Facilitating Translational Research

Event Details

Meeting Objectives

• Discuss mechanisms that initiate and drive progression of glomerular diseases to End Stage Kidney Disease.
• Explore Targets and Pathways to therapeutic development for each glomerular disease.
• Assess existing biomarkers that define the diagnosis, initiation, progression, and/or relapse of glomerular disease.
• Discuss approaches to cooperation among industry, academia, government, and non-nephrology researchers who deal with other organ systems.

Agenda

April 17, 2012

7:00 a.m. - 8:00 a.m.

Registration

8:00 a.m. - 8:15 a.m.

Introduction:
Dr. Griffin Rodgers, Dr. Robert Star and Dr. Michael Flessner

Opening Remarks and Objectives:
Dr. Robert Star and Dr. Michael Flessner

8:00 a.m. - 8:15 a.m.

Introduction:
Dr. Gregory Germino, Deputy Director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Opening Remarks and Objectives
Dr. Robert Star, Director, Division of Kidney, Urologic, and Hematologic Diseases, and
Dr. Michael Flessner, NIDDK

8:15 a.m. - 9:15 a.m.

State of the Art Lectures (20 minutes each followed by 10 minute discussion)
Moderator: Dr. Charles Alpers, University of Washington

• Challenges in Therapy Development for Rare Diseases: Cystic Fibrosis
  Dr. Frank Accurso, Children's Hospital Colorado
• Lessons from lupus rrials/network
  Dr. Neil Solomons, Vifor Pharma
• Glomerular Disease: Recent Travels and the Road Ahead
  Dr. William Couser, University of Washington

9:15 a.m. - 9:45 a.m.

Break

9:45 a.m. - 12:00 p.m.

Disease-Specific Pathophysiologic Mechanisms and Translational Tools (to answer the question: Where are we? with emphasis on therapeutic targets and Potential diagnostic, prognostic, and alternative outcome biomarkers/diagnostic tests) (15 minutes each followed by 10 minute discussion)
Moderator: Dr. Roger Wiggins, University of Michigan

• Minimal change/FSGS: Genetics of Primary Podocytopathies
  Dr. Friedhelm Hildebrandt, University of Michigan
• Minimal change/FSGS: Mechanisms and Biomarkers for Primary Podocytopathies
  Dr. Jochen Reiser, University of Miami
• Membranous
  Dr. David Salant, Boston University
• IgA Nephropathy
  Dr. Jan Novak, University of Alabama
• Vasculitis
  Dr. Ron Falk, University of North Carolina
• C3 Glomerular Disease
  Dr. Terry Cook, Imperial College

12:00 p.m. - 1:00 p.m.

Lunch (bag lunch in breakout rooms)

1:00 p.m. - 3:00 p.m.

Outcomes for Translational and Clinical Research: Patient Reported Outcomes (PROs), Biomarkers, Role of Registry (15 minutes each followed by 5 minute discussion)
Moderator: Dr. Michael Flessner, NIDDK

• Clinical Trial Outcome Assessments (symptoms = patient reported outcomes, particularly in pediatrics) in glomerular diseases
  Dr. Elektra Papadopoulos, U.S. Food and Drug Administration (FDA)
• Use of Data Standards and Disease Models in PKD. Why do this prospectively?
  Dr. Ron Perrone, Tufts University
• Qualification of diagnostic or severity/stratification biomarkers
  Dr. Courtney Lias, FDA
• Possible bases of approval for progression or relapse of glomerular diseases
  Dr. Aliza Thompson, FDA
• CKD Biomarker Consortium: Sample requirements for discovery and reproducibility of renal biomarkers
  Dr. Jennifer Van Eyk, The Johns Hopkins University
• Pros, Cons, Pitfalls of a Biological Repository
  Dr. Elizabeth Wagner, National Heart, Lung, and Blood Institute

3:00 p.m. - 3:15 p.m.

Break

3:15 p.m. - 5:00 p.m.

Breakout sessions: These will begin on Day 1 and carry forward to Day 2.

Topics for individual breakout sessions:

1. Minimal Change/FSGS
   Dr. Bill Smoyer, Nationwide Children's Hospital, and Dr. Jochen Reiser, University of Miami
2. Membranous
   Dr. David Salant, Boston University, Dr. Fernando Fervenza, Mayo Clinic, and Dr. Daniel Cattran, University of Toronto
3. IgA Nephropathy
   Dr. Heather Reich, University of Toronto, and Dr. Jan Novak, University of Alabama
4. Vasculitis
   Dr. Peter Merkel, University of Pennsylvania
5. C3 Glomerular Disease
   Dr. Terry Cook, Imperial College, and Dr. Gerry Appel, Columbia University
6. Cross-Cutting Data standards and alternative outcome biomarkers (proteinuria, podocyte products, quantitative pathology, functional data, etc?) needed by all glomerular diseases?
   Dr. Larry Holzman, University of Pennsylvania, Dr. Roger Wiggins, University of Michigan, and Dr. Agnes Fogo, Vanderbilt University

Goals for breakout groups:

1. Establish targets for future research in each disease.
2. Provide specific suggestions on moving the field forward.

Discussion questions for breakout groups:

1. What are the key questions to be answered for this disease? Can we define the critically important next steps?
2. Can we use mechanistic knowledge of the disease to identify specific biological targets? Are there biomarkers for diagnosis, onset, severity, progression, or relapse of the disease?
3. What steps are needed to qualify specific biomarkers for specific purposes?
4. What cross-cutting tools are needed across all of these diseases (data standards, common alternative outcome biomarkers, partnerships, etc.)?
5. What is needed in a disease registry to facilitate therapeutic development? Can this be obtained using existing clinical EHRs (HMOs, VA, and large health care consortia)?
6. How does the nephrology community partner with industry, advocacy groups, and government?

5:00 p.m. - 6:00 p.m.

Preliminary Reports from the Breakout Sessions and General Discussion

6:00 p.m.

Adjourn - Day 1

April 18, 2012

7:00 a.m. - 8:00 a.m.

Registration

8:00 a.m. - 10:00 a.m.

Perspective From Industry: Drug Development for Glomerular Diseases
Moderator: Paul Kimmel, NIDDK

Suggested participants :
Dr. Frank Dieterle, Novartis
Dr. Paul Brunetta, MD Genentech: Phase III Planning for Novel Therapies in Autoimmune Glomerulonephritis - An Industry Perspective
Dr. Loghman-Adham and Dr. Scheiflinger: An Anti-Macrophage Migration Inhibitory Factor Antibody for Treatment of Lupus Nephritis
Dr. Matt Breyer, Lilly
Dr. Tom Daniels, Celgene
Dr. Tracy McGowan J and J, Centocor
Dr. Maria Bobadilla, Roche
Dr. Glenn Reinhart, Boehringer-Ingelheim (may send substitute)
Dr. Yuuichi Tomura, Astellas
Dr. Camille Bedrosian, Alexion
Dr. Alan Cohen, Interimmune
Dr. Dennis Andress, Abbott
Dr. Steve Ledbetter, Genzyme

Industry Panel Discussion - Panel of Speakers and Moderator (30 minutes)

10:00 a.m. - 10:30 a.m.

Break

10:30 a.m. - 12:30 p.m.

Registry/Collaboration Roundtable: What have we learned from other diseases and existing glomerular disease registries? What are the critical issues? (10 minutes each followed by 5 minute discussion)
Moderator: Dr. William Schnaper, Northwestern University

• Office of Rare Diseases, NIH
  Dr. Stephen Groft
• Public-Private Partnerships/Collaborative Opportunities/An NIH View
  Dr. Barbara Mittleman
• UNC Glomerular Disease Collaborative Network
  Dr. Patrick Nachman, University of North Carolina
• Toronto Glomerulonephritis Registry Group
  Dr. Dan Cattran, University of Toronto
• Lessons from NEPTUNE/biopsy registries/recruitment challenges
  Dr. Matthias Kretzler, University of Michigan
• European Registry for Glomerular Diseases
  Dr. Charles Pusey, Imperial College
• Nephcure
  Dr. Larry Holzman, University of Pennsylvania
• Would a consortium of clinical EHRs (such as the proposed HMO-collaboration in the "Health Care Systems Collaboratory") coupled with "cloud computing" have more utility than registries?
  Dr. John Sedor, Case Western University

12:30 p.m. - 3:00 p.m.

Breakout Sessions (Lunch in Breakout sessions) Topics for individual breakout sessions : same as Day 1

3:00 p.m. - 4:30 p.m.

Reports from the Breakout Sessions: Discussion and Next Steps

4:30 p.m. - 5:00 p.m.

Summary -Dr. Robert Star and Dr. Michael Flessner, NIDDK

5:00 p.m.

Adjournment