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.

In this ARC TALKS webinar, Dr. Heather Landau discussed current treatment options for relapsed and refractory AL amyloidosis patients.

Dr. Landau also talked about the current testing methodology used to monitor a patient’s disease status, and the role maintenance therapy may play in the management of relapsed AL amyloidosis.

In addition to describing considerations that may factor into recommended treatment plans for patients, Dr. Landau shared details on novel therapies being evaluated in clinical trials.

0:00 - Introduction
3:15 - Relapsed / Refractory AL Amyloidosis Overview
20:31 - Managing Relapsed / Refractory AL Amyloidosis
38:27 - Q&A
39:03 - How do patients know if they're heading towards relapsed / refractory AL amyloidosis?
41:47 - How often do you monitor for relapsed AL amyloidosis?
42:57 - What is "minimal residual disease?"
46:54 - How common is relapsed AL amyloidosis?
48:52 - Is Venetoclax a therapy option?
50:28 - Alternatives after Daratumumab?
52:38 - How important is increased screening?
55:01 - How are light-chain and M spike levels correlated?
56:45 - Kappa Lambda FLC vs dFLC ratios
59:53 - Conclusion

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.

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

Dr. Anita D’Souza, associate professor of hematology and medical oncology from the Medical College of Wisconsin, discusses recent study findings regarding the timing and co-occurrence of symptoms within the three years prior to a diagnosis of light chain (AL) amyloidosis. Organized by organ system, Dr. D’Souza lists red-flag symptoms that patients may experience, typically presenting in varying combinations. Analyzing EHR records she sought to understand whether red-flag symptoms were indeed being identified, and how their occurrence would accumulate over time towards diagnosis.

Bottom line: 
* This work confirms that patients are being diagnosed within the healthcare system with multiple red-flag diagnoses before AL amyloidosis is formally diagnosed.
* It is possible to catalog these diagnoses from electronic health records data and thus has the potential for earlier diagnosis of this complex disease.
* This study shows the timing and combinations between these diagnoses and lays the foundation to develop clinical algorithms aimed at earlier recognition of AL amyloidosis.

The Institute for Future Health held a hybrid conference on January 14th, 2023. The focus was on expanding the knowledge of cardiac amyloidosis amongst Arizona clinicians. This event was one of the first exclusively dedicated to cardiac amyloidosis for local physicians across multiple healthcare professions. Cardiac amyloidosis is a disease of protein misfolding that leads to congestive heart failure and disabling neuropathy. Cardiac amyloidosis can be caused by transthyretin or light-chain amyloidosis. The pace of knowledge in this field is greatly accelerating, and it is important for clinicians, nurses, research scientists, and patients to be aware of new developments.

Learn more @ theifh.org

*This activity is supported by an independent medical educational grant from Pfizer, Inc.

This 5th part is devoted to amyloid neuropathy. The focus will be on AL amyloidosis (Amyloid light chain) and ATTR amyloidosis (Amyloid transthyretin protein or TRR protein).

- Dr. Tanya STOJKOVIC, France (Clinician)
- Prof. David ADAMS, France (Clinician)
- Jean-Christophe FIDALGO, (Patient testimony)

#EuropeanReferenceNetwork #ERNs #EURONMD #NMD #PERIPHERALNEUROPATHY #Amyloidosis

Nir Pillar, MD, PhD, a postdoctoral fellow at the University of California, Los Angeles, received a 2023 PhRMA Foundation Postdoctoral Fellowship in Translational Medicine for his research combining novel microscopy methodologies and artificial intelligence to improve detection of systemic amyloidosis. Watch this video to learn about Pillar and his research.