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

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Mathew Maurer, MD
Cardiologist and Medical Director, The HCM Center at NewYork-Presbyterian Hospital/Columbia University Medical Center
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Vaishali Sanchorawala, MD
Director, Amyloidosis Center, Boston University School of Medicine
BIO+

Speakers

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Raymond Comenzo, MD
Director, Transfusion Services; Director, John C. Davis Myeloma and Amyloid Program; Professor, Tufts University School of Medicine
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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+
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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+
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Melanie Blank, MD
Division of Clinical Evaluation and Pharmacology/Toxicology in the Office of Tissues and Advanced Therapies, CBER, US Food and Drug Administration
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Michelle Campbell, PhD
Senior Clinical Analyst, Division of Neurology Products (DNP),CDER, US Food and Drug Administration
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Nicole Gormley, MD
Division Director for the Division of Hematologic Malignancies II, CDER, US Food and Drug Administration
BIO+
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Jessica Lee
Associate Director, Division of Gastroenterology and Inborn Errors Products (DGIEP), US Food and Drug Administration
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Kimberly Smith, MD
Nephrology, Division of Cardiovascular and Renal Products (DCRP), US Food and Drug Administration
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Isabelle Lousada
Founder and CEO, Amyloidosis Research Consortium
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Jonathan Cohen
AL Amyloidosis patient
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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:

Recordings

The following meeting recordings are hosted on the FDA website.