Current status and prospect of anti-amyloid fibril therapy in AL amyloidosis

Jinghua Wang; Jian Li; Liye Zhong

doi:10.1016/j.blre.2024.101207

Current status and prospect of anti-amyloid fibril therapy in AL amyloidosis

Blood Rev. 2024 Apr 17:101207. doi: 10.1016/j.blre.2024.101207. Online ahead of print.

Authors

Jinghua Wang¹, Jian Li², Liye Zhong³

Affiliations

¹ Department of Hematology, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, China.
² Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China. Electronic address: lijian@pumch.cn.
³ Department of Hematology, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, China. Electronic address: liye_zhong@hotmail.com.

PMID: 38692939
DOI: 10.1016/j.blre.2024.101207

Abstract

Amyloid light-chain (AL) amyloidosis is a rare hematological disease that produces abnormal monoclonal immunoglobulin light chains to form amyloid fibrils that are deposited in tissues, resulting in organ damage and dysfunction. Advanced AL amyloidosis has a very poor prognosis with a high risk of early mortality. The combination of anti-plasma cell therapy and amyloid fibrils clearance is the optimal treatment strategy, which takes into account both symptoms and root causes. However, research on anti-amyloid fibrils lags far behind research on anti-plasma cells, and there is currently no approved treatment that could clear amyloid fibrils. Nevertheless, anti-amyloid fibril therapies are being actively investigated recently and have shown potential in clinical trials. In this review, we aim to outline the preclinical work and clinical efficacy of fibril-directed therapies for AL amyloidosis.

Keywords: Anti-fibril therapy; Doxycycline; Light-chain amyloidosis; Monoclonal antibody; Pathogenesis; Stabilizer.

Publication types

Review