Potentiating CD20 monoclonal antibody therapy by targeting complement C3 fragments covalently deposited on lymphoma cells

S Sivasubramanian Baskar (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) H Haiyong Peng E Erika M. Gaglione (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) E Elizabeth J. Carstens M Margaret A. Lindorfer (3Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA) I Inhye E. Ahn (1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) S Sarah E. M. Herman (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) M Martin Skarzynski (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) J Jing Chang K Keyvan Keyvanfar (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) V Vicent Butera (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) A Amy Blackburn (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) B Bérengère Vire (1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) I Irina Maric (5Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD) M Maryalice Stetler-Stevenson (5Flow Cytometry Unit, Laboratory of Pathology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD) C Constance M. Yuan (2Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD) M Michael A. Eckhaus (6Division of Veterinary Resources, Office of Research Services, National Institutes of Health, Bethesda, MD) S Susan Soto (1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) M Mohammed Z. H. Farooqui (1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD) R Ronald P. Taylor (3Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA) C Christoph Rader A Adrian Wiestner (1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD)

Abstract

Abstract Monoclonal antibodies (mAbs) improve survival of patients with mature B-cell malignancies. Fcγ receptor–dependent effector mechanisms kill tumor cells but can promote antigen loss through trogocytosis, contributing to treatment failures. Cell-bound mAbs trigger the complement cascade to deposit C3 activation fragments and lyse cells. Within 24 hours after ofatumumab administration to patients with chronic lymphocytic leukemia (CLL), circulating tumor cells had lost CD20 and were opsonized with C3d, the terminal covalently bound form of complement protein C3. We hypothesized that C3d provides a target to eliminate residual CD20− tumor cells. To test this hypothesis, we generated C8xi, a mouse/human chimeric immunoglobulin G1 (IgG1) that reacts with human but not mouse C3d. C8xi was effective in a patient-derived xenograft model against CD20−, C3d opsonized CLL cells from patients treated with ofatumumab. We also generated rabbit mAbs, 2 of which were chosen because they bound mouse and human C3d with low nanomolar affinity but were minimally cross-reactive with full-length C3. Anti-C3d rabbit/human chimeric IgG1 in combination with ofatumumab or rituximab prolonged survival of xenografted mice that model 3 different types of non-Hodgkin lymphoma (NHL). For example, in a diffuse large B-cell lymphoma model (SU-DHL-6), median survival with single-agent CD20 mAb was 114 days but was not reached for mAb combination treatment (P = .008). In another NHL model (SU-DHL-4), single-agent and combination mAb therapy eradicated lymphoma in most mice. In long-term survivors from both cohorts, there was no evidence of adverse effects. We propose that C3d mAbs combined with complement-fixing CD20 mAbs can overcome antigen-loss escape and increase efficacy of mAb-based therapy.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 12
Published March 20, 2025
Pages 1309-1320
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (22)

S

Sivasubramanian Baskar

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

H

Haiyong Peng

E

Erika M. Gaglione

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

E

Elizabeth J. Carstens

M

Margaret A. Lindorfer

3Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA

I

Inhye E. Ahn

1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

S

Sarah E. M. Herman

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

M

Martin Skarzynski

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

J

Jing Chang

K

Keyvan Keyvanfar

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

V

Vicent Butera

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

A

Amy Blackburn

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

B

Bérengère Vire

1Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

I

Irina Maric

5Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD

M

Maryalice Stetler-Stevenson

5Flow Cytometry Unit, Laboratory of Pathology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD

C

Constance M. Yuan

2Laboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, MD

M

Michael A. Eckhaus

6Division of Veterinary Resources, Office of Research Services, National Institutes of Health, Bethesda, MD

S

Susan Soto

1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

M

Mohammed Z. H. Farooqui

1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

R

Ronald P. Taylor

3Department of Biochemistry and Molecular Genetics, University of Virginia School of Medicine, Charlottesville, VA

C

Christoph Rader

A

Adrian Wiestner

1Laboratory of Lymphoid Malignancies, Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD