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Review
. 2025 Aug:73:101299.
doi: 10.1016/j.blre.2025.101299. Epub 2025 May 9.

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

Anam Ashfaque  1 , Yara Shatnawi  2 , Shahzad Raza  3 , Diana Basali  4 , Jack Khouri  5 , Louis Williams  6 , Sandra Mazzoni  7 , Christy Samaras  8 , Hamza Hassan  9 , Utkarsh Acharya  10 , Chakra Chaulagain  11 , Faiz Anwer  12
Affiliations
Free article
Review

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

Anam Ashfaque et al. Blood Rev. 2025 Aug.
Free article

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.

Keywords: AL amyloidosis; Amyloid fibril-directed therapy; Anselamimab; Anti-fibril therapy; CAEL-101; Light chain amyloidosis.

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Conflict of interest statement

Declaration of Competing Interest Shahzad Raza: received an honorarium as an advisory board for Pfizer, Prothena Biosciences, and KiTE pharma; received research funding from Janssen, Naxcella Inc., Poseida Therapeutics. Jack Khouri: Janssen-Honoraria, Membership on an entity's Board of Directors or advisory committees, Other: Consultant; GPCR Therapeutics, Inc.-Honoraria; Prothena-Honoraria; Legend-Membership on an entity's Board of Directors or advisory committees. Louis Williams: Honoraria- Janssen, Bristol Myers Squibb; Research Funding-Abbvie Inc. Sandra Mazzoni: consulting relationship with Janssen and J &J Consulting. Faiz Anwer: served as an advisor and speaker for Bristol Myers Squibb, Celgene, Caribou biosciences and Poseida Therapeutics; received research funding from Allogene Therapeutic, Celgene, GlaxoSmithKline, Bristol Myers Squibb, and Caribou Biosciences. The remaining authors do not have any personal, financial, or professional conflicts of interest related to the contents discussed in this manuscript.

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