Suppression to removal, an emerging therapeutic approach for AL amyloidosis: A comprehensive review with early human data and pharmacokinetics of CAEL-101 antibody

Department

Internal Medicine

Document Type

Article

Publication Title

Blood Reviews

Abstract

Light chain (AL amyloidosis) is a rare disorder characterized by the deposition of misfolded light chains in various organs, causing progressive organ damage. Current therapeutic agents do not remove amyloid aggregates already present in the organs, which are the major determinants of morbidity and mortality. Therefore, drugs targeting amyloid fibrils are currently being investigated. This article provides a comprehensive review of a novel, fibril-directed antibody, CAEL-101 (Anselamimab), which removes fibrillary aggregates from the organs, restoring organ function. Overall, CAEL-101 has demonstrated a favorable toxicity profile and improved organ responses, with faster treatment response time than current therapies in phase I/II trials, and is expected to have promising outcomes in the ongoing phase III studies. Combining anti-plasma cell dyscrasia (suppression) and fibril-directed agents (removal) is a novel therapeutic approach, providing optimism for organ function recovery and enhanced quality of life and survival, especially in patients with severe diseases.

First Page

101299

DOI

10.1016/j.blre.2025.101299

Volume

73

Publication Date

8-1-2025

Medical Subject Headings

Humans; Immunoglobulin Light-chain Amyloidosis; Antibodies, Monoclonal, Humanized; Animals; Amyloid; Antibodies, Monoclonal

PubMed ID

40382293

Link to Full Text

Share

COinS