Understanding AL Amyloidosis
November 2019
The inaugural meeting of the Amyloidosis Forum took place in November 2019, in the Great Room at the FDA. This meeting established the priorities for the Forum. To learn more about the priorities read The Amyloidosis Forum: A public private partnership to advance drug development in AL amyloidosis.
Faculty
Moderators
Speakers
Raymond Comenzo, MD
Director, Transfusion Services; Director, John C. Davis Myeloma and Amyloid Program; Professor, Tufts University School of Medicine
BIO+
Angela Dispenzieri, MD
Consultant; Division of Hematology; Serene M. and Frances C. Durling Professor of Medicine and of Laboratory Medicine and Pathology; Hematology Research Chair – Mayo Clinic College of Medicine. President of the International Society of Amyloidosis
BIO+
Preston Dunnmon, MD, MBA, FACP, FACC
Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology, CDER, US Food and Drug Administration
BIO+
Melanie Blank, MD
Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies, CBER, US Food and Drug Administration
Michelle Campbell, PhD
Senior Clinical Analyst, Division of Neurology Products (DNP),CDER, US Food and Drug Administration
BIO+
Nicole Gormley, MD
Division Director for the Division of Hematologic Malignancies II, CDER, US Food and Drug Administration
BIO+
Jessica Lee
Associate Director, Division of Gastroenterology and Inborn Errors Products (DGIEP), US Food and Drug Administration
Kimberly Smith, MD
Nephrology, Division of Cardiovascular and Renal Products (DCRP), US Food and Drug Administration
Jonathan Cohen
AL Amyloidosis patient
Matt Millen
AL Amyloidosis patient and former Detroit Lions team president
Agenda
Moderators: Mat Maurer, MD PhD
9:00 am
Welcome
Isabelle Lousada
9:05 am
Opening Remarks
Preston Dunnmon, MD
9:15 am
Pathobiology and Clinical Features
Raymond Comenzo, MD
9:35 am
Epidemiology
Angela Dispenzieri, MD
9:45 am
Study Populations and Staging Systems
Angela Dispenzieri, MD
10:15 am
Panel Discussion
11:20 am
Quality of Life
Vaishali Sanchorawala, MD
11:30 am
Panel Discussion
11:40 am
FDA Approach to Multisystemic Diseases
Preston Dunnmon, MD
12:10 pm
Q+A
Moderators: Vaishali Sanchorawala, MD
1:20 pm
Patient Perspectives
Isabelle Lousada and 2 patients
1:40 pm
Challenges Designing Trials with Newly Diagnosed Patients
Ashutosh Wechalekar, MBBS, MD, FRCP, FRCPath, DM
1:55 pm
Challenges Designing Trials with Relapsed/Refractory Patients
Heather Landau, MD
2:10 pm
Panel Discussion
3:10 pm
Roadmap for Future Discussions
Preston Dunnmon
3:40 pm
Closing Remarks
Isabelle Lousada and Preston Dunnmon
Publications
The following publications resulted from the meeting:
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The Amyloidosis Forum: a public-private partnership to advance drug development in AL amyloidosis
Lousada and The Inaugural Amyloidosis Forum Panelists, Orphanet Journal of Rare Diseases (2020) 15:268
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The Amyloidosis Forum: A Public-Private Partnership to Advance Drug Development in AL Amyloidosis
Isabelle Lousada, Vaishali Sanchorawala, Angela Dispenzieri, Kristen Hsu, Robyn Himick, Preston Dunnmon, Mathew Maurer, International Society of Amyloidosis, Annual Meeting 2020