Treatment of AL amyloidosis in the era of novel immune and cellular therapies Review


Authors: Sarubbi, C.; Abowali, H.; Varga, C.; Landau, H.
Review Title: Treatment of AL amyloidosis in the era of novel immune and cellular therapies
Abstract: Light chain (AL) amyloidosis is a plasma cell disorder distinguished from multiple myeloma (MM) by the degree of organ involvement due to tissue deposition of misfolded proteins. Treatments for AL amyloidosis have largely been borrowed from those developed for patients with MM. High-dose chemotherapy followed by autologous stem cell transplant (ASCT) has historically been associated with the best outcomes. The recent incorporation of daratumumab into up front therapy represents a significant advance and has changed the treatment paradigm, calling into question the role of ASCT. The development of very active novel immune and cellular therapies, specifically B cell maturation antigen (BCMA)-directed therapies, has similarly been transformative for patients with MM and is now being studied in patients with AL amyloidosis. These include chimeric antigen receptor (CAR) T cells, bispecific antibodies, and antibody drug conjugates. Although limited, preliminary data in patients with relapsed and refractory AL amyloidosis are showing promising results, and it is expected that the treatment landscape for AL amyloidosis will continue to evolve. Particular attention to safety, potential for organ recovery, and quality of life will be important when evaluating new treatments and/or treatment paradigms. Copyright © 2024 Sarubbi, Abowali, Varga and Landau.
Keywords: side effect; drug megadose; multiple myeloma; phase 2 clinical trial; stem cell transplant; plasma cell; chimeric antigen receptor; cell therapy; short survey; phase 3 clinical trial; phase 1 clinical trial; drug therapy; adverse drug reaction; therapy; longitudinal study; oral drug administration; drug comparison; al amyloidosis; bispecific antibody; human; bispecific antibodies; b cell maturation antigen; daratumumab; antibody drug conjugate; chimeric antigen receptor t-cell immunotherapy; car t cell therapy; chimeric antigen receptor t-cell
Journal Title: Frontiers in Oncology
Volume: 14
ISSN: 2234-943X
Publisher: Frontiers Media S.A.  
Date Published: 2024-06-28
Start Page: 1425521
Language: English
DOI: 10.3389/fonc.2024.1425521
PROVIDER: scopus
PMCID: PMC11239377
PUBMED: 39007104
DOI/URL:
Notes: Review -- MSK Cancer Center Support Group (P30 CA008748) acknowledged in PubMed and PDF -- MSK corresponding author is Heather Landau -- Source: Scopus
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  1. Heather Jolie Landau
    419 Landau