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Review
. 2017 Dec:180:129-138.
doi: 10.1016/j.pharmthera.2017.06.011. Epub 2017 Jun 22.

Novel pharmacotherapies for cardiac amyloidosis

Kevin M Alexander  1 , Avinainder Singh  1 , Rodney H Falk  2
Affiliations
Review

Novel pharmacotherapies for cardiac amyloidosis

Kevin M Alexander et al. Pharmacol Ther. 2017 Dec.

Abstract

Amyloidosis refers to a range of protein misfolding disorders that can cause organ dysfunction through progressive fibril deposition. Cardiac involvement often leads to significant morbidity and mortality and increasingly has been recognized as an important cause of heart failure. The two main forms of cardiac amyloidosis, light chain (AL) and transthyretin (ATTR) amyloidosis, have distinct mechanisms of pathogenesis. Recent insights have led to the development of novel pharmacotherapies with the potential to significantly impact each disease. This review will summarize the preclinical and clinical data for these emerging treatments for AL and ATTR amyloidosis.

Keywords: Amyloidosis; Cardiomyopathy; Light chain; Neuropathy; Therapy; Transthyretin.

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Conflict of interest statement

Conflict of interest statement

Dr. Alexander has received an investigator-initiated research grant from Pfizer. Dr. Singh has nothing to declare. Dr. Falk serves as a consultant for Ionis Pharmaceuticals and Alnylam Pharmaceuticals and has received research support from GlaxoSmithKline.

Figures

Fig. 1
Fig. 1
Therapeutic Targets for Amyloidosis. Treatment for AL amyloidosis focuses on eliminating the bone marrow-derived B cell clone responsible for producing toxic light chains. ATTR therapies aim to significantly reduce hepatic production of TTR or to stabilize the native TTR homotetramer to prevent dissociation into proamyloidogenic monomers. Several drugs have been developed to disrupt and clear already deposited AL and/or TTR amyloid fibrils.
See this image and copyright information in PMC

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