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Disease-Modifying Pharmacological Therapies for Transthyretin Cardiac Amyloidosis

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Abstract

Transthyretin-related cardiac amyloidosis is a disease that is poorly understood and challenging to manage; however, pharmacotherapeutic developments in recent years has raised awareness and promoted earlier diagnosis. Transthyretin protein is a thyroid hormone-binding protein which transports thyroxine from the bloodstream to the brain, and cardiac involvement is either acquired or hereditary. This protein can misfold and lead to abnormal fibril formation, causing the extracellular deposition of ATTR into the heart. Further fibril deposition leads to irreversible organ dysfunction. Disease-modifying pharmacological therapies can be divided into three categories: ATTR stabilizers (diflunisal, tafamidis, and AG10), ATTR silencers (patisiran and inotersen), and ATTR disruptors (doxycycline and ursodeoxycholic acid and polyphenol (-)-epigallocatechin gallate). This manuscript reviews these emerging pharmacological treatments available and under study for ATTR cardiac amyloidosis.

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Authors and Affiliations

  1. Heart Failure/Transplantation, Division of Cardiology, The Pauley Heart Center, Virginia Commonwealth University, MCV Campus, P.O. Box 980204, Richmond, VA, 23298-0204, USA

    Jay H. Park, Laura F. Cei & Keyur B. Shah

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  1. Jay H. Park
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Correspondence to Keyur B. Shah.

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KBS is a consultant for Akcea Therapeutics and Alnylam Pharmaceuticals. KBS serves on the ATTRIBUTE Trial Steering Committee for Eidos Therapeutics.

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Park, J.H., Cei, L.F. & Shah, K.B. Disease-Modifying Pharmacological Therapies for Transthyretin Cardiac Amyloidosis. SN Compr. Clin. Med. 2, 1607–1613 (2020). https://doi.org/10.1007/s42399-020-00420-y

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