| Systemic amyloidosis MOD

A few excerpts from a 3-minute MOD animation that provides an insightful overview of amyloidosis, a group of rare and devastating progressive diseases caused by the accumulation of misfolded proteins.

The most common form of systemic amyloidosis is amyloid light chain (AL) amyloidosis, which is caused by the misfolding of immunoglobulin light chains produced by abnormal plasma cells in the bone marrow. These misfolded light chains aggregate, forming amyloid fibrils that accumulate in organs and tissues, leading to damage and dysfunction.

A crucial detail and task for us was to create an accurate depiction of the immunoglobulin light chain monomer, its conformational misfolding – and ultimately, its aggregation and assembly into amyloid fibrils – which accumulate into deposits and damage organs.

Given the significant impact on the heart (may lead to conditions such as cardiomyopathy in over 75% of patients), the focus of this animation is on illustrating the damage caused by amyloid deposits in cardiac tissue. While the heart is most commonly affected, amyloid fibrils can also accumulate in other organs such as the liver, wrists, hands, feet, tongue, and gastrointestinal tract.

By illustrating these key processes, this animation highlights the severe impact of amyloid deposits on the heart and other organs affected by AL amyloidosis.

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Recording of the Amyloidosis Support Groups webinar on Cardiac Amyloidosis in AL (light chain) and ATTR (transthyretin) Amyloidosis from January 20, 2024. This webinar recording has been edited for content and time.

Moderator:
Muriel Finkel, President, Amyloidosis Support Groups

Speakers:
Melissa Lyle, MD, FACC, Assistant Professor of Medicine, Cardiology Lead, Multidisciplinary Amyloid Clinic, Mayo Clinic, Jacksonville, FL
Justin Grodin, MD, MPH, FACC, Associate Professor of Medicine, Medical Director, Internal Medicine Clinical Trials Unit, University of Texas Southwestern Medical Center, Dallas, Texas
Barry Trachtenberg, MD, FACC, Advanced Heart Failure Cardiology, Houston Cardiovascular Associates, J.C. Walter Transplant Center at Houston Methodist Hospital, Houston, TX
Paula Schmitt, Executive Director, Amyloidosis Support Groups
*Gustavo Buchele, MD, PhD, Senior Director, Clinical Development, Alexion Pharmaceuticals, Boston, MA
*Mark McGovern, RN, CCRN, Vice President Clinical Operations, Eidos Therapeutics, a subsidiary of BridgeBio Pharma, San Francisco, CA
*Joy Olbertz, PharmD, PhD, Senior Director, Clinical Scientist, Intellia Therapeutics, Inc., Cambridge, MA

Chapters
0:00 Welcome
2:53 Speaker Introductions
3:52 ATTR and AL Treatment Charts
4:34 Melissa Lyle: Cardiac Amyloidosis: Heart Failure Symptoms and Treatment
32:23 Justin Grodin: Arrhythmias, Pacemakers, & Defibrillators in Cardiac Amyloidosis
1:00:05 Barry Trachtenberg: Advanced Heart Failure Therapies in Cardiac Amyloidosis
1:32:27 Panel Discussion/Q&A
1:57:54 Paula Schmitt: Financial Assistance
2:07:39 Panel Discussion/Q&A cont’d
2:11:13 Pharma Trial Information
2:11:43 Panel Comments on Pharmaceutical Trials

*Live webinar only. Presentations and commentary from these speakers were authorized for the live webinar only and are excluded from this video recording.

Presented by the Amyloidosis Speakers Bureau ... Learn about wild-type amyloidosis from both an expert clinician and his patient. Listen to world-renowned cardiologist Dr. Mathew S. Maurer of New York-Presbyterian Hospital / Columbia University Medical Center overview wild-type amyloidosis (ATTRwt), followed by one of his patients John Basdavanos as he shares his journey with wild-type amyloidosis.
(Based on highlights from a presentation to the Northeast Ohio Medical University College of Medicine.)

Dr. Morie Gertz, professor of medicine at the Mayo Clinic in Rochester and world renowned expert in amyloidosis, talks about how the immune system can be harnessed in the fight against amyloidosis and multiple myeloma. He discusses two immune-directed therapies: CAR-T and Bispecific Antibodies. Dr. Gertz eloquently, and in an easy-to-understand way, summarizes the goal of immune-directed therapies and the two approaches today, including the processes, outcomes, advantages, and risks to be considered. This is a must-watch video for physicians from a legendary expert.

One of the organs most frequently impacted by amyloidosis is the kidney. Dr. Jeffrey Zonder from the Karmanos Cancer Institute provides a brief summary of amyloidosis and the most common types of this disease. From there, he goes on to describe how AL (Light Chain) and AA ((Apo)serum Amyloid A) are the two most prevalent types to affect the kidneys, detail how amyloidosis affects the kidneys, how this damage is assessed, and strategies for reducing kidney damage.

An animated video produced for Astra Zeneca about Transthyretin Amyloidosis (ATTR), a progressive and debilitating disorder that is caused by unstable TTR protein that form amyloid fibrils, which deposit in organs and tissues, especially the peripheral nervous system and heart.

Produced by Link Studio (a board certified medical illustration and animation studio) and BioScience Communications for Astra Zeneca.

Medical Animation, Storyboard, Art Style: Mike Linkinhoker, MA, CMI / Link Studio LLC
Art Direction, Script: BioScience Communications

Beyond Standard of Care Treatment for Light Chain (AL) Amyloidosis: The Promise of Tomorrow
Presented by Dr. Heather Landau.
Heather Landau, MD discusses standard and emerging immunotherapies for AL Amyloidosis and how the treatment landscape has changed based on recent advances. She will describe novel immunotherapies including antibody-drug conjugates, bispecific antibodies, and chimeric antigen receptor (CAR) T cells and their potential role in AL amyloidosis. Dr. Landau will review other novel therapies undergoing clinical investigation including those that directly target amyloid.

A follow-up from the March 12th National AL-Amyloidosis Patient-to-Provider Connection Forum.