amyloidosis forum

Workshop Panelists

Considerations for Novel Endpoint Development in AL Amyloidosis

  January 22nd, 2021

11:00am-3:30pm EST

Faculty

 

PPP Hosts

Isabelle Lousada

Isabelle Lousada
CEO and President, Amyloidosis Research Consortium

Isabelle Lousada has been the driving force behind ARC, building successful collaborations and programs across the sectors to advance the science and understanding of the amyloidosis diseases. She was diagnosed with AL amyloidosis in 1996 and for the past twenty years she has been committed to empowering other patients while serving on several boards and committees, speaking at leadership meetings and key events to encourage research, increase access, and support the critical and unmet needs of amyloidosis patients. She currently serves as co-chair for the regulatory science working group at the EveryLife Foundation.

Preston Dunnmon FDA

Preston Dunnmon MD, MBA, FACP, FACC
Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology US Food and Drug Administration

Dr. Dunnmon is the acting Medical Team Leader in the Division of Cardiology and Nephrology as part of the Office of New Drugs (OND), Office of Drug Evaluation I (ODEI), Division of Cardiovascular and Renal Products (DCRP) within the Center for Drug Evaluation and Research (CDER), to serve as the CDER Public Private Partnership (PPP) Liaison to ARC.

Moderator

Sarah Cairns-Smith

Sarah Cairns Smith, PhD, MBA
Chair, Amyloidosis Research Consortium

Sarah Cairns-Smith is a Senior Advisor at The Boston Consulting Group (BCG) where she was a Senior Partner and Managing Director. She was also a Harvard University Advanced Leadership Initiative Fellow in 2019. At BCG she was a leader in the social impact, organization and health care global practices and led BCG’s Scientist Network, a group of over 500 PhD/MDs. She specialized in innovation-related consulting with a strong emphasis on R&D and launch stage projects and novel technologies. Her technology interests span biotech and biopharma through digital. Sarah has consulted across the private, public and social sectors including to several of the world’s major foundations, multi-lateral organizations and leading companies. Sarah’s work in developing economies while consulting with the United Nations, Bill & Melinda Gates Foundation and USAID led to a focus on the impact, funding and governance of digital in economic development. Sarah has deep expertise in the development of innovation-related consortia and public-private partnerships, for instance through helping to establish the Digital Impact Alliance (DIAL) and the National Institute of Health’s Accelerating Medicines Partnership.Sarah chairs the Board of the Amyloidosis Research Consortium and is a member of the Board of Directors for Engender Health. She holds a BSc. in Microbiology and Genetics from University College London. Sarah received her PhD in Biochemistry from the Imperial Cancer Research Fund, London (Francis Crick Institute) and was a post-doctoral researcher at the Center for Neurobiology, Columbia University. Sarah also holds an MBA from Columbia Business School (Distinction).

Working Group Leaders

Hematology Working Group

Angela Dispenzieri

Angela Dispenzieri, MD
Professor of Medicine and of Laboratory Medicine and Pathology, Mayo Clinic Rochester

Angela Dispenzieri, M.D., is a consultant in the Division of Hematology, Department of Internal Medicine at Mayo Clinic, and serves as Chair of Hematology Research. She has joint appointments in the Division of Clinical Biochemistry and Immunology, Department of Laboratory Medicine and Pathology, and the Department of Molecular Medicine. Dr. Dispenzieri holds the academic rank of Professor of Medicine and of Laboratory Medicine and Pathology, Mayo Clinic College of Medicine, and she is recognized with the distinction of a named professorship, the Serene M. and Frances C. Durling Professorship.

Cardiac Working Group

Mathew Maurer

Mathew S. Maurer, MD
Cardiologist and Medical Director, The HCM Center at NewYork-Presbyterian Hospital/Columbia University Medical Center
Dr. Maurer directs the Clinical Cardiovascular Research Laboratory for the Elderly (CCRLE) at the Allen Hospital of NewYork-Presbyterian Hospital. Dr. Maurer is a member of the Advanced Cardiac Care Center at NewYork-Presbyterian/Columbia. He has extensive expertise in amyloidosis and has served as a principle investigator in clinical trials for hereditary amyloidosis.

 

Renal Working Group

Nelson Leung, MD, 
Consultant, Professor of Medicine, Mayo Clinic, Rochester
Dr. Leung joined the staff at the Mayo clinic in the division of Nephrology and Hypertension, he later joined the Division of Hematology and holds a joint appointment in both divisions. His main interest is in monoclonal gammopathy related kidney diseases. He has written extensively on renal amyloidosis, myeloma cast nephropathy and monoclonal immunoglobulin deposition disease. He introduced the concept of monoclonal gammopathy of renal significance and has made substantial contributions in the treatment of paraprotein related kidney diseases. He is outgoing president of the International Kidney and Monoclonal Gammopathy Research Group.

 

Nerve, GI and Liver Working Group

Michelle Mauermann

Michelle Mauermann, MD
Consultant, Department of Neurology, Professor of Neurology, Mayo Clinic, Rochester
Dr. Mauermann is a neurology consultant with expertise in peripheral neuropathies at Mayo Clinic in Rochester, MN. A Professor of Neurology and board certified in Neurology, Electrodiagnostic Medicine, Neuromuscular Medicine and Neuromuscular Pathology. In clinical practice, Dr. Mauermann provides care to a variety of peripheral nerve and neuromuscular conditions. She has special interests in paraproteinemic neuropathies, neoplastic neuropathies and treatment related toxicity, peripheral nerve tumors and nerve pathology. Her research focuses on elucidating the mechanisms of paraproteinemic neuropathies.

 

Health Related Quality of Life Working Group

Vaishali Sanchorawala, MD
Professor of Medicine. Director, Amyloidosis Center, Boston University School of Medicine and Boston Medical Center

Dr. Sanchorawala is the Professor of Medicine and Director of the Amyloidosis Center of Boston University School of Medicine and Boston Medical Center. A pioneer in the field of clinical research in AL amyloidosis. Her work in the treatment of AL amyloidosis has been published in many peer-reviewed journals, which has resulted in the evolution of the standard of care for these patients. She is currently heading several clinical trials in the treatment of AL amyloidosis. She serves on the executive steering committee of the Amyloidosis Research Consortium, the board of the International Society of Amyloidosis as secretary, and as an associate editor of Amyloid, Journal of Protein Folding Disorders.

Statistical Working Group

James Signorovitch, PhD
Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
Dr. Signorovitch advises life sciences companies on data analytics for business critical research and decision making. He has broad experience leading the strategic development and implementation of analytics across the product life cycle, from early-phase clinical studies to market access and real-world evidence generation. Dr. Signorovitch’s practice areas span trial design, multi-stakeholder collaborations, natural history studies, regulatory interactions, health economic modeling, global reimbursement submissions, policy evaluation, real-world evidence development, individualized medicine, predictive analytics, and due diligence for acquisitions. He has particular expertise in developing and applying new methodologies to address health care research challenges, and in designing analytics platforms to enhance collaborative research and decision making. Dr. Signorovitch’s work has been used to inform clinical regulators and health care payers in US and global markets, published in peer-reviewed journals, and presented at clinical and economic research conferences. Prior to joining Analysis Group, Dr. Signorovitch was a research fellow at the Harvard-MIT Division of Health Sciences and Technology.

Working Group Members

Hematology Working Group
  • 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
  • Shaji Kumar, MD, Mark and Judy Mullins Professor of Hematological Malignancies Consultant, Division of Hematology Professor of Medicine
  • Ashutosh Wechalekar, MBBS, MD, FRCP, FRCPath, DM, Professor of Medicine and Haematology, Hon. Consultant, National Amyloidosis Centre, University College London (Royal Free Campus); Dept of Haematology, Royal Free Hospital; Dept of Haematology, University College Hospital, London
  • Nicole Gormley, MD,  Division Director for the Division of Hematologic Malignancies II, CDER,  US  Food and Drug Administration
  • Beatriz Flores, MD, Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
  • Brendan Weiss, MD, Senior Director Multiple Myeloma Strategy Leader, Janssen Research and Development
  • Liang Xiu, PhD, Director, Biostatistics, Janssen Pharmaceuticals
  • James Signorovitch, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
  • Mackenzie Boedicker, Founder, President, and Chairman of the Board, Mackenzie’s Mission
Cardiac Working Group
  • Mathew S. Maurer, MD, Medical director of The HCM Center at New York-Presbyterian Hospital/Columbia University Medical Center
  • Claudio Rapezzi, MD Professor of Cardiology, University of Ferrara, Italy
  • Marianna Fontana, MD, PhD, Professor of Cardiology, Hon. Consultant Cardiologist Deputy clinical and academic lead for the National Amyloidosis Centre, UCL Cardiac CMR service, University College London (Royal Free Campus)
  • Ronald Witteles, MD Professor of Medicine (Cardiovascular Medicine, Co-Director, Stanford Amyloid Center, Co-Director, Stanford Multidisciplinary Sarcoidosis Program Director, Internal Medicine Residency Training Program, Stanford University School of Medicine
  • Giampaolo Merlini, MD, Director of the Center for Research and Treatment of Systemic Amyloidosis, and of the Biotechnology Research Laboratories, Scientific Institute Policlinico San Matteo, University of Pavia, Italy
  • Preston Dunnmon, MD, MBA, FACP, FACC, Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology, CDER, US Food and Drug Administration
  • Krishna Prasad, MD, Senior Medical Assessor Medicines and Healthcare products Regulatory Agency (MHRA), UK
  • Cristina Quarta, MD, PhD, Director, Clinical Development Sciences, Global Medical Lead for Cael-101 and AG10 programs, Metabolic Diseases Therapeutic Area, Alexion
  • James Signorovitch, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
  • Isabelle Lousada, CEO and President, Amyloidosis Research Consortium
Renal Working Group
  • Nelson Leung, MD, Consultant, Department of Nephrology, Professor of Medicine, Mayo Clinic College of Medicine  
  • Andrea Harvasi, MD, Assistant Professor of Medicine Boston University Medical Center Department of Medicine/Renal Section/Amyloidosis Center
  • Julian Gillmore, MBBS, MD, PhD, FRCP, FRCPath, Professor of Medicine & Honorary Consultant Nephrologist Head, Centre for Amyloidosis & Acute Phase Proteins Research Lead, National Amyloidosis Centre, UCL Division of Medicine
  • Raymond Comenzo, MD, Director, Transfusion Services; Director, John C. Davis Myeloma and Amyloid Program; Professor, Tufts University School of Medicine
  • Kimberly Smith, MD, MS, Clinical Team Leader, Division of Cardiology and Nephrology (DCN), CDER, US Food and Drug Administration
  • Krishna Prasad, MD, Senior Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
  • Yolanda Barbachano, Senior Statistician, Medicines and Healthcare products Regulatory Agency (MHRA), UK
  • Spencer Guthrie, MBA, MPH , CEO, Attralus, Inc
  • James Signorovitch, PhD,  Managing Principal, Analysis Group, Inc., Health Economics and Outcomes Research, Epidemiology and Biostatistics
  • Dena Heath, Northern California Support Group and Amyloidosis Research Consortium
Nerve, GI and Liver Working Group
  • Michelle Mauermann, Consultant, Department of Neurology, Professor of Neurology, Mayo Clinic Rochester
  • John Clarke, MD, Director, Esophageal Program, Stanford University School of Medicine, Stanford University
  • William Litchy, MD, Consultant in Neurology, Department of Neurology, Mayo Clinic Rochester
  • Morie A Gertz, MD, M.A.C.P., Consultant, Division of Hematology, Roland Seidler Jr. Professor Department of Medicine, College of Medicine, Mayo Distinguished Clinician|
  • Laura Obici, MD, Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy
  • 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
  • Laura Jawidzik, MD, Clinical Team Lead, Division of Neurology 1, CDER, US Food and Drug Administration
  • Matthew Kowalik, MD, Acting Team Leader, Division of Gastroenterology, CDER, US Food and Drug Administration
  • Sarrit Kovacs, PhD, Clinical Reviewer, Division of Gastroenterology, CDER, US Food and Drug Administration
  • Douglas Faller, MD, PhD, Executive Medical Director, Takeda
  • James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
  • Yolanda Barbachano, Senior Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK
  • Kathy Koontz, Patient Representative
HQRoL Working Group
  • Vaishali Sanchorawala, MD, Professor of Medicine, Director, Amyloidosis Center, Boston University School of Medicine and Boston Medical Center
  • Anita D’Souza, MD,  Associate Professor of Medicine, Scientific Director, CIBMTR® Medical College of Wisconsin, Froedtert Clinical Cancer Center
  • Elektra Papadopolous, MD, Acting Director Division of Clinical Outcome Assessment, Office of New Drugs, CDER, US Food and Drug Administration
  • Michelle Campbell, PhD, Senior Clinical Analyst, Division of Neurology Products (DNP), CDER, US Food and Drug Administration
  • Beatriz Flores, MD, Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA), UK
  • Tiffany Quock, Senior Medical Director, Prothena Biosciences Inc
  • Avery Rizio, PhD – Scientist QualityMetric Incorporated, LLC
  • James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
  • Paula Schmitt, Executive Director, Amyloidosis Support Groups
  • Michelle White, PhD, Vice President and Senior Scientist, QualityMetric
Statistical Working Group
  • James Signorovitch, PhD, Managing Principal, Health Economics and Outcomes Research, Epidemiology and Biostatistics, Analysis Group, Inc.
  • Preston Dunnmon, MD, MBA, FACP, FACC – Cardiologist, Medical Team Leader (acting) in the Division of Cardiology and Nephrology, CDER, US Food and Drug Administration
  • Mark Rothmann, PhD,  Deputy Division Director, Division of Biometrics II, CDER, US Food and Drug Administration
  • Jialu Zhang, PhD,  Division of Biometrics II, CDER, US Food and Drug Administration
  • David Brown, Expert Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK
  • Yolanda Barbachano,  Senior Statistician, Medicines and Healthcare Products Regulatory Agency (MHRA), UK