Developing Therapy for Transthyretin Amyloidosis

Courtney M Campbell; Kathleen Zhang; Daniel J Lenihan; Ronald Witteles

doi:10.1016/j.amjmed.2022.01.002

Developing Therapy for Transthyretin Amyloidosis

Am J Med. 2022 Apr:135 Suppl 1:S44-S48. doi: 10.1016/j.amjmed.2022.01.002. Epub 2022 Jan 22.

Authors

Courtney M Campbell¹, Kathleen Zhang², Daniel J Lenihan², Ronald Witteles³

Affiliations

¹ Cardio-Oncology Center of Excellence, Washington University in St. Louis, Mo. Electronic address: campbellc@wustl.edu.
² Cardio-Oncology Center of Excellence, Washington University in St. Louis, Mo.
³ Stanford Amyloid Center, Division of Cardiovascular Medicine, Stanford University School of Medicine, Calif.

PMID: 35077703
DOI: 10.1016/j.amjmed.2022.01.002

Abstract

Transthyretin amyloidosis (ATTR) is an under-recognized cause of cardiomyopathy and neuropathy. Until recently, there were limited therapeutic options for ATTR. However, new therapeutics, including tafamidis, patisiran, and inotersen, increase both quality and length of life in patients with ATTR. This review details the chronological development of ATTR therapies through landmark clinical trials. In addition, we discuss emerging ATTR therapies including improvements in drug delivery methods, antibodies to break down deposited amyloid fibrils, and gene editing. ATTR is a prime example of how an understanding of the pathophysiological basis of disease can lead to effective therapies. The future of ATTR therapy is bright, with every reason to believe outcomes will continue to improve.

Keywords: ATTR; Cardiomyopathy; Inotersen; Neuropathy; Patisiran; Tafamidis.

Publication types

Review

MeSH terms

Amyloid Neuropathies, Familial* / drug therapy
Amyloid Neuropathies, Familial* / genetics
Cardiomyopathies* / drug therapy
Humans
Prealbumin / genetics

Substances

Prealbumin

Supplementary concepts

Amyloidosis, Hereditary, Transthyretin-Related