Antibody-based immunotherapy for treatment of immunoglobulin light-chain amyloidosis.
AL amyloidosis
CAR-T cells
immunotherapy
monoclonal antibodies
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
12 2020
12 2020
Historique:
received:
27
01
2020
revised:
05
04
2020
accepted:
07
04
2020
pubmed:
17
4
2020
medline:
24
3
2021
entrez:
17
4
2020
Statut:
ppublish
Résumé
Immunoglobulin light-chain (AL) amyloidosis is a clonal plasma cell disorder characterised by production and deposition of misfolded monoclonal light chains in vital organs with potential to cause irreversible organ damage. The treatment of AL amyloidosis has evolved along the lines of multiple myeloma (MM) owing to clonal plasma cells being at the root of both disease processes. Treatment with melphalan and autologous haematopoietic cell transplantation, as well as proteasome inhibitors and immunomodulatory agents, are the standard of care for AL amyloidosis. While these treatment modalities are highly effective against the neoplastic plasma cells, patients often relapse and those with advanced disease may be unable to tolerate these treatments due to side-effects. Immunotherapy with monoclonal antibodies, bispecific antibodies, antibody-drug conjugates and chimeric antigen receptor T cells have revolutionised the treatment armamentarium for MM. These novel immunotherapy agents are in the early phases of evaluation and clinical development for patients with AL amyloidosis. The present review aims to discuss the role of novel immunotherapies currently in development and their potential for use in the treatment of AL amyloidosis.
Substances chimiques
Antibodies, Bispecific
0
Antibodies, Monoclonal
0
Antineoplastic Agents, Immunological
0
Immunoconjugates
0
Immunoglobulin Light Chains
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
673-681Informations de copyright
© 2020 British Society for Haematology and John Wiley & Sons Ltd.
Références
Maurer MS, Elliott P, Comenzo R, Semigran M, Rapezzi C. Addressing common questions encountered in the diagnosis and management of cardiac amyloidosis. Circulation. 2017;135:1357-77.
Bhat A, Selmi C, Naguwa SM, Cheema GS, Gershwin ME. Currents concepts on the immunopathology of amyloidosis. Clin Rev Allergy Immunol. 2010;38:97-106.
Merlini G, Bellotti V. Molecular mechanisms of amyloidosis. N Engl J Med. 2003;349:583-96.
Kyle RA, Linos A, Beard CM,Linke RP, Gertz MA, O'Fallon WM, et al. Incidence and natural history of primary systemic amyloidosis In Olmsted County, Minnesota, 1950 through 1989. Blood. 1992;79:1817-22.
Quock TP, Yan T, Chang E, Guthrie S, Broder MS. Epidemiology of AL amyloidosis: a real world study using US claims data. Blood Adv. 2018;2:1046-53.
Kumar S, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Colby C, et al. Revised prognostic staging system for light chain amyloidosis incorporating cardiac biomarkers and serum free light chain measurements. J Clin Oncol. 2012;30:989-95.
Vaxman I, Gertz M. Recent advances in the diagnosis, risk stratification, and management of systemic light-chain amyloidosis. Acta Haematol. 2019;141:93-106.
Muchtar E, Dispenzieri A, Leung N, Lacy MQ, Buadi FK, Dingli D, et al. Depth of organ response in AL amyloidosis is associated with improved survival: grading the organ response criteria. Leukemia. 2018;32:2240-9.
Muchtar E, Gertz MA, Kumar SK, Lacy MQ, Dingli D, Buadi FK, et al. Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death. Blood. 2017;129:2111-9.
Cordes S, Dispenzieri A, Lacy MQ, Hayman SR, Buadi FK, Dingli D, et al. Ten-year survival after autologous stem cell transplantation for immunoglobulin light chain amyloidosis. Cancer. 2012;118:6105-9.
Gertz MA, Lacy MQ, Dispenzieri A, Kumar SK, Dingli D, Leung N, et al. Refinement in patient selection to reduce treatment-related mortality from autologous stem cell transplantation in amyloidosis. Bone Marrow Transplant. 2013;48:557-61.
Lebovic D, Hoffman J, Levine BM, Hassoun H, Landau H, Goldsmith Y, et al. Predictors of survival in patients with systemic light-chain amyloidosis and cardiac involvement initially ineligible for stem cell transplantation and treated with oral melphalan and dexamethasone. Br J Haematol. 2008;143:369-73.
Kumar SK, Hayman SR, Buadi FK, Roy V, Lacy MQ, Gertz MA, et al. Lenalidomide, cyclophosphamide, and dexamethasone (CRd) for light-chain amyloidosis: long-term results from a phase 2 trial. Blood. 2012;119:4860-7.
Palladini G, Sachchithanantham S, Milani P, Gillmore J, Foli A, Lachmann H, et al. A European collaborative study of cyclophosphamide, bortezomib, and dexamethasone in upfront treatment of systemic AL amyloidosis. Blood. 2015;126:612-5.
Lee JY, Lim SH, Kim SJ, Lee GY, Lee JE, Choi JO, et al. Bortezomib, melphalan, and prednisolone combination chemotherapy for newly diagnosed light chain (AL) amyloidosis. Amyloid. 2014;21:261-6.
Mikhael JR, Schuster SR, Jimenez-Zepeda VH, Bello N, Spong J, Reeder CB, et al. Cyclophosphamide-bortezomib-dexamethasone (CyBorD) produces rapid and complete hematologic response in patients with AL amyloidosis. Blood. 2012;119:4391-4.
Cibeira MT, Oriol A, Lahuerta JJ, Mateos MV, de la Rubia J, Hernández MT, et al. A phase II trial of lenalidomide, dexamethasone and cyclophosphamide for newly diagnosed patients with systemic immunoglobulin light chain amyloidosis. Br J Haematol. 2015;170:804-13.
Manwani R, Hegenbart U, Mahmood S, Sachchithanantham S, Kyriakou C, Yong K, et al. Deferred autologous stem cell transplantation in systemic AL amyloidosis. Blood Cancer J. 2018;8:101.
Tapan U, Seldin DC, Finn KT, Fennessey S, Shelton A, Zeldis JB, et al. Increases in B-type natriuretic peptide (BNP) during treatment with lenalidomide in AL amyloidosis. Blood. 2010;116:5071-2.
Jaccard A, Comenzo RL, Hari P, Hawkins PN, Roussel M, Morel P, et al. Efficacy of bortezomib, cyclophosphamide and dexamethasone in treatment-naïve patients with high-risk cardiac AL amyloidosis (Mayo Clinic stage III). Haematologica. 2014;99:1479-85.
Bochtler T, Hegenbart U, Cremer FW, Heiss C, Benner A, Hose D, et al. Evaluation of the cytogenetic aberration pattern in amyloid light chain amyloidosis as compared with monoclonal gammopathy of undetermined significance reveals common pathways of karyotypic instability. Blood. 2008;111:4700-5.
Bochtler T, Hegenbart U, Kunz C, Granzow M, Benner A, Seckinger A, et al. Translocation t(11; 14) is associated with adverse outcome in patients with newly diagnosed AL amyloidosis when treated with bortezomib-based regimens. J Clin Oncol. 2015;33:1371-8.
Tandon N, Sidana S, Gertz MA, Dispenzieri A, Lacy MQ, Buadi FK, et al. Treatment patterns and outcome following initial relapse or refractory disease in patients with systemic light chain amyloidosis. Am J Hematol. 2017;92:549-54.
Franssem LE, Mutis T, Lohorst HM, van de Donk NW. Immunotherapy in myeloma: how far have we come? Ther Adv Hematol. 2019;10:1-19.
Lin P, Owens R, Tricot G, Wilson CS. Flow cytometric immunophenotypic analysis of 306 cases of multiple myeloma. Am J Clin Pathol. 2004;121:482-8.
Sanchez L, Wang Y, Siegel DS, Wang ML. Daratumumab: a first-in-class CD38 monoclonal antibody for the treatment of multiple myeloma. J Hematol Oncol. 2016;9:51.
Dimopoulos MA, Oriol A, Nahi H, San-Miguel J, Bahlis NJ, Usmani SZ, et al. Daratumumab, lenalidomide, and dexamethasone for multiple myeloma. N Engl J Med. 2016;375:1319-31.
Palumbo A, Chanan-Khan A, Weisel K, Nooka AK, Masszi T, Beksac M, et al. Daratumumab, bortezomib, and dexamethasone for multiple myeloma. N Engl J Med. 2016;375:754-66.
Seckinger A, Hillengass J, Emde M, Beck S, Kimmich C, Dittrich T, et al. CD38 as immunotherapeutic target in light chain amyloidosis and multiple myeloma-Association with molecular entities, risk, survival, and mechanisms of upfront resistance. Front Immunol. 2018;9:1676.
Sher T, Fenton B, Akhtar A, Gertz MA. First report of safety and efficacy of daratumumab in 2 cases of advanced immunoglobulin light chain amyloidosis. Blood. 2016;128:1987-9.
Kaufman GP, Schrier SL, Lafayette RA, Arai, S, Witteles RM, Liedtke M, Daratumumab yields rapid and deep hematologic responses in patients with heavily pretreated AL amyloidosis. Blood. 2017;130:900-2.
Kimmich C, Schönland, SO, Müller-Tidow C, Dittrich T, Hegenbart U, Daratumumab monotherapy in forty-eight heavily pretreated patients with advanced systemic light-chain amyloidosis. The XVIth International Symposium on Amyloidosis; March 26-29, 2018; Kumamoto, Japan: International Society of Amyloidosis; 2018.
Abeykoon JP, Zanwar S, Dispenzieri A, Gertz MA, Leung N, Kourelis T, et al. Daratumumab-based therapy in patients with heavily-pretreated AL amyloidosis. Leukemia. 2019;33:531-6.
Chung A, Kaufman GP, Sidana S, Eckhert E, Schrier SL, Lafayette, RA, et al. Organ responses with daratumumab therapy in previously treated AL amyloidosis. Blood Adv. 2020;4:458-66.
Lecumberri R, Krsnik I, Askari E, Sirvent M, González-Pérez MS, Escalante F, et al.Treatment with daratumumab in patients with relapsed/refractory AL amyloidosis: a multicentric retrospective study and review of the literature. Amyloid. 2020 [Epub ahead of print]. https://doi.org/10.1080/13506129.2020.1730791.
Roussel M, Merlini G, Chevret S, Arnulf B, Stoppa, AM, Perrot A, et al.A prospective phase II of daratumumab in previously treated systemic light-chain (AL) amyloidosis patients. Blood. 2020 [Epub ahead of print]. https://doi.org/10.1182/blood.2019004369.
Sanchorawala V, Sarosiek S, Schulman A, Mistark M, Migre ME, Cruz R, et al.Safety, Tolerability, and Response Rates of Daratumumab in Relapsed AL Amyloidosis: Results of a Phase II Study. Blood. 2020 [Epub ahead of print]. https://doi.org/10.1182/blood.2019004436.
Deckert J, Wetzel MC, Bartle LM. SAR650984, a novel humanized CD38-targeting antibody, demonstrates potent antitumor activity in models of multiple myeloma and other CD38+ hematologic malignancies. Clin Cancer Res. 2014;20:4574-83.
Martin T, Baz R, Benson DM. A phase 1b study of isatuximab plus lenalidomide and dexamethasone for relapsed/refractory multiple myeloma. Blood. 2017;129:3294-303.
Hsi ED, Steinle R, Balasa B. CS1, a potential new therapeutic antibody target for the treatment of multiple myeloma. Clin Cancer Res. 2008;14:2775-84.
Lonial S, Dimopoulos M, Palumbo A. Elotuzumab therapy for relapsed or refractory multiple myeloma. N Engl J Med. 2015;373:621-31.
Dimopoulos MA, Dytfeld D, Grosicki S, Moreau P, Takezako N, Hori M, et al. Elotuzumab plus pomalidomide and dexamethasone for multiple myeloma. N Engl J Med. 2018;379:1811-22.
Iqbal SM, Stecklein K, Sarow J, Krabak M, Hillengass J, McCarthy P. Elotuzumab in combination with lenalidomide and dexamethasone for treatment-resistant immunoglobulin light chain amyloidosis with multiple myeloma. Clin Lymphoma Myeloma Leuk. 2019;19:e33-6.
Wall JS, Kennel SJ, Williams A, Richey T, Stuckey A, Huang Y, et al. AL amyloid imaging and therapy with a monoclonal antibody to a cryptic epitope on amyloid fibrils. PLoS ONE. 2012;7:e52686.
Gertz MA, Landau H, Comenzo RL, Seldin D, Weiss B, Zonder J, et al. First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction. J Clin Oncol. 2016;34:1097-103.
Prothena Therapeutics Ltd. Prothena Discontinues Development Of NEOD001 For AL Amyloidosis. Available at: http://ir.prothena.com/news-releases/news-release-details/prothena-discontinues-development-neod001-al-amyloidosis. [updated April 23, 2018].
Solomon A, Weiss DT, Wall JS. Therapeutic potential of chimeric amyloid-reactive monoclonal antibody 11-1F4. Clin Cancer Res. 2003;9:3831s-3838s.
Wall JS, Kennel S, Stuckey A, Long MJ, Townsend DW, Smith G, et al. Radioimmunodetection of amyloid deposits in patients with AL amyloidosis. Blood. 2010;116:2241-4.
Edwards CV, Gould J, Langer AL, Mapara M, Radhakrishnan J, Maurer MS, et al. Interim analysis of the phase 1a/b study of chimeric fibril-reactive monoclonal antibody 11-1F4 in patients with AL amyloidosis. Amyloid. 2017;24(Suppl. 1):58-9.
Pepys MB, Booth DR, Hutchinson WL, Gallimore JR, Collins IM, Hohenester E. Amyloid P component. A critical review. Amyloid. 1997;4:274-95.
Pepys MB, Herbert J, Hutchinson WL, Tennent GA, Lachmann HJ, Gallimore JR, et al. Targeted pharmacological depletion of serum amyloid P component for treatment of human amyloidosis. Nature. 2002;417:254-9.
Gillmore JD, Tennent GA, Hutchinson WL, Gallimore JR, Lachmann HJ, et al. Sustained pharmacological depletion of serum amyloid P component in patients with systemic amyloidosis. Br. J. Haematol. 2010;148:760-7.
Richards DB, Cookson LM, Berges AC, Barton SV, Lane T, Ritter JM, et al. Therapeutic Clearance of Amyloid by Antibodies to Serum Amyloid P Component. N Engl J Med. 2015;373:1106-14.
Richards DB, Cookson LM, Barton SV, Liefaard L, Lane T, Hutt DF, et al. Repeat doses of antibody to serum amyloid P component clear amyloidosis deposits in patient with systemic amyloidosis. Sci Transl Med. 2018;10:eaan3128. https://doi.org/10.1126/scitranslmed.aan3128
June CH, Sadelain M. Chimeric antigen receptor therapy. N Engl J Med. 2018;379:64-73.
Raje N, Berdeja J, Lin Y, Siegel D, Jagannath S, Madduri D, et al. Original article anti-BCMA CAR T-cell therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019;380:1726-37.
Rosenzweig M, Urak R, Walter M, Lim L, Sanchez JF, Krishnan A, et al. Preclinical data support leveraging CS1 chimeric antigen receptor T-cell therapy for systemic light chain amyloidosis. Cytotherapy. 2017;19:861-6.
O'Neal J, Ritchey JK, Cooper M, Niswonger J, Ghobadi A, DiPersio JF. CS1 targeted chimeric antigen receptors (CAR) for treatment of multiple myeloma. Biol Blood Marrow Transplant. 2019;25:s382-3.
Wang XL, Walter M, Urak R, Weng L, Huynh C, Lim L, et al. Lenalidomide enhances the function of CS1 chimeric antigen receptor-redirected T cells against multiple myeloma. Clin Cancer Res. 2018;24:106-19.
Zhang Y, Chen L, Wang Y, Li X, Hughes T, Klingemann H, et al. Combination therapy with daratumumab and CAR-NK targeting CS1 for multiple myeloma. Blood. 2016;128:1342.
Bal S, Sigler A, Chan A, Chung D, Dogan A, Giralt S, et al. BCMA expression in AL amyloidosis. Clin Lymphoma Myeloma Leuk. 2019;19 (Suppl.):e306.
Godara A, Shou P, Rosenthal B, Kugelmass A, Toskic D, Fogaren T, et al. B-Cell maturation antigen (BCMA) in systemic light-chain amyloidosis (AL): association with disease activity and its modulation with gamma-secretase inhibition. Blood. 2019;134(Suppl.1):4409.
Sanchez E, Li M, Kitto A, Li J, Wang CS, Kirk DT et al. Serum B-cell maturation antigen is elevated in multiple myeloma and correlates with disease status and survival. Br J Haematol. 2012;158:727-38.
Godara A, Zhou, P., Kugelmass, A, Ma X, Rosenthal B, Toskic D, et al. Presence of soluble and cell-surface B-cell maturation antigen in systemic light-chain amyloidosis and its modulation by gamma-secretase inhibition. Am J Hematol. 2020;95: E110-3
Timmers M, Roez G, Wang Y, Campillo-Davo D. Chimeric antigen receptor-modified T-Cell therapy in multiple myeloma: beyond B cell maturation antigen. Front. Immunol. 2019;10:1613.
Chen KH, Wada M, Pinz KG, Liu H, Shuai X, Chen X, et al. A compound chimeric antigen receptor strategy for targeting multiple myeloma. Leukemia. 2018;32:402-12.
Teachey DT, Lacey SF, Shaw PA, Melenhorst JJ, Maude SL, Frey N, et al. Identification of predictive biomarkers for cytokine release syndrome after chimeric antigen receptor T-cell therapy for acute lymphoblastic leukemia. Cancer Discov. 2016;6:664-79.
Ganatra S, Carver JR, Hayek SS, Ky B, Leja MJ, Lenihan DJ, et al. Chimeric antigen receptor T-cell therapy for cancer and heart: JACC council perspectives. J Am Coll Cardiol. 2019;74:3153-63.
Yu B, Liu D. Antibody-drug conjugates in clinical trials for lymphoid malignancies and multiple myeloma. J Hematol Oncol. 2019;12:94.
Tai YT, Mayes PA, Acharya C, Zhong MY, Cea M, Cagnetta A, et al. Novel anti-B-cell maturation antigen antibody-drug conjugate (GSK2857916) selectively induces killing of multiple myeloma. Blood. 2014;123:3128-38.
Tudel A, Lendavi N, Popat R, Voorhees PM, Reeves B, Libby EN, et al. Antibody-drug conjugate, GSK2857916, in relapsed/refractory multiple myeloma: an update on safety and efficacy from dose expansion phase I study. Blood Cancer J. 2019;9:37.
Lonial S, Lee HC, Badros A, Trudel S, Nooka AK, Chari A, et al. Belantamab mafodotin for relapsed or refractory multiple myeloma (DREAMM-2): a two-arm, randomised, open-label, phase 2 study. Lancet Oncol. 2020;21:P207-21.
Ailawadhi S, Kelly KR, Vescio RA, Jagannath S, Wolf J, Gharibo M, et al. A phase I study to assess the safety and pharmacokinetics of single-agent lorvotuzumab mertansine (IMGN901) in patients with relapsed and/or refractory CD-56-positive multiple myeloma. Clin Lymphoma Myeloma Leuk. 2019;19:29-34.
Govindan SV, Cardillo TM, Sharkey RM, Tat F, Gold DV, Goldenberg DM. Milatuzumab-SN-38 Conjugates for the Treatment of CD74 Cancers. Mol Cancer Ther. 2013;12:968-78.
Jagannath S, Heffner LT Jr, Ailawadhi S, Munshi NC, Zimmerman TM, Rosenblatt J. et al. Indatuximab ravtansine (BT062) monotherapy in patients with relapsed and/or refractory multiple myeloma. Clin Lymphoma Myeloma Leuk. 2019;19:372-80.
Berdeja JG, Hernandez-Ilizaliturri F, Chanan-Khan AA, Patel M, Kelly KR, Running KL, et al. Phase I study of lorvotuzumab mertansine (LM, IMGN901) in combination with lenalidomide (Len) and dexamethasone (Dex) in patients with CD56-positive relapsed or relapsed/refractory multiple myeloma (MM). Blood. 2012;120:728.
Schonfeld K, Zuber C, Pinkas J, Hader T, Bernoster K, Uherek C. Indatuximab ravtansine (BT062) combination treatment in multiple myeloma: pre-clinical studies. J Hematol Oncol. 2017;10:13.
Kelly KR, Siegel DS, Chanan-Khan AA, Somlo G, Heffner LT, Jagannath S, et al. Indatuximab ravtansine (BT062) in combination with low-dose dexamethasone and lenalidomide or pomalidomide: clinical activity in patients with relapsed /refractory multiple myeloma. Blood. 2016;128:4486.
Panowski SH, Kuo T, Chen A, Geng T, Van Blarcom TJ, Lindquist K, et al. Preclinical evaluation of a potent anti-BCMA CD3 bispecific molecule for the treatment of multiple myeloma. Blood. 2016;128:383.
Lesokhin AM, Raje NS, Gasparetto C, Walker J, Krupka HI, Joh T, et al. Phase I, open-label study to evaluate the safety, pharmacokinetic, pharmacodynamic, and clinical activity of PF-06863135, a B-cell maturation antigen/CD3 bispecific antibody, in patients with relapsed/refractory advanced multiple myeloma. Blood. 2018;s3229.
Pillarisetti K, Baldwin E, Babich A, Majewski N, Barone L, Li Y, et al. Development of a New BCMAxCD3 Duobody® antibody for multiple myeloma. Blood. 2016;128:2116.
Hipp S, Tai YT, Blanset D, Liu J, Yu T, Wen K, et al. A novel BCMA/CD3 bispecific T-cell engager for the treatment of multiple myeloma induces selective lysis in vitro and in vivo. Leukemia. 2017;31:2278.
Cho SF, Lin L, Xing L, Liu J, Yu T, Wen K, et al. Anti-BCMA BiTE® AMG 701 potently induces specific T cell lysis of human multiple myeloma (MM) cells and immunomodulation in the bone marrow microenvironment. Blood. 2018;132(Suppl. 1):592.
Costa LJ, Wong S, Bermudez A, de la Rubia J, Mateos MV, Ocio EM, et al. First clinical study of the B-Cell maturation antigen (BCMA) 2+1 T cell engager (TCE) CC-93269 in patients (Pts) with relapsed/refractory multiple myeloma (RRMM): interim results of a phase 1 multicenter trial. Blood. 2019;134(Suppl. 1):143.
Zuch de Zafra CL, Fajardo F, Zhong W, Bernett MJ, Muchhal US, Moore GL, et al. Targeting multiple myeloma with AMG 424, a novel anti-CD38/CD3 bispecific T-cell-recruiting antibody optimized for cytotoxicity and cytokine release. Clin Cancer Res. 2019;25:3921-33.
Richter JR, Landgren CO, Kauh JS, Back J, Salhi Y, Reddy V, et al. Phase 1, multicenter, open-label study of single-agent bispecific antibody t-cell engager GBR 1342 in relapsed/refractory multiple myeloma. J Clin Oncol. 2018;36:15.
Thorsten RP, Uwe R, Susanne W, Haneke T, Klausz K, et al. Preclinical characterization of AFM26, a novel B cell maturation antigen (BCMA)-directed tetravalent bispecific antibody for high affinity retargeting of NK cells against myeloma. Blood. 2018;132 (Suppl. 1):1927.
Development of anti-amyloid fibril-reactive monoclonal antibodies for treatment of AL amyloidosis. https://www.cumc.columbia.edu/labs/lentzsch-lab/projects/development-anti-amyloid-fibril-reactive-monoclonal-antibodies-treatment-al-amyloidosis. Accessed 14 January 2020.