Targeting cell-surface VISTA expression on allospecific naïve T cells promotes tolerance

B Brent H. Koehn (1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN) E Elizabeth C. Nowak (2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH) S Sladjana Skopelja-Gardner A Asim Saha (1University of Minnesota, Division of Blood and Marrow Transplantation, Department of Pediatrics, Minneapolis, United States) M Michael C. Zaiken (1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN) J Jeremy Allred (1University of Minnesota, Division of Hematology, Oncology and Transplantation, Minneapolis, United States) Y Yiyung Peng (1Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN) W Wilson L. Davis (2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH) I Isabelle Le Mercier (4AvenCell Therapeutics Inc, Watertown, MA) N Nicholas Schwertner (5Lifordi Immunotherapeutics, Burlington, MA) M Michael J. Molloy (6ImmuNext Inc, Alton, NH) J Jay Rothstein (4AvenCell Therapeutics Inc, Watertown, MA) C Catherine Carriere (4AvenCell Therapeutics Inc, Watertown, MA) M Megan J. Riddle (1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN) C Cindy R. Eide (1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN) J Jakub Tolar (1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN) A Angela Panoskaltsis-Mortari K Kyle D. Smith (1Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN) K Keli L. Hippen (1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN) T Tae Kon Kim (1Vanderbilt University Medical Center, Division of Hematology/Oncology, Department of Medicine, Nashville, United States) R Randolph J. Noelle (2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH) B Bruce R. Blazar (Department of Pediatrics, Division of Blood & Marrow Transplant & Cellular Therapy, University of Minnesota)

Abstract

Abstract The success of allogeneic hematopoietic stem cell transplantation (allo-HSCT) can be limited by graft-versus-host disease (GVHD). T-cell activation is a key factor in GVHD progression. Costimulatory signals can be counterbalanced by coinhibitory signals such as the checkpoint molecule VISTA (V-domain immunoglobulin-containing suppressor of T-cell activation)/programmed death-1 homolog that restrains activation and maintains donor T-cell quiescence. A single dose of anti-VISTA monoclonal antibody (mAb) prevents acute GVHD lethality in multiple models. Naïve donor T cells express moderate VISTA levels, which transiently increase in allo-HSCT recipients in association with T-cell receptor signaling, leading to heightened susceptibility to anti-VISTA mAb–mediated depletion, in contrast to donor T cells transferred to syngeneic recipients. Anti-VISTA mAb donor T-cell depletion was compatible with rapamycin but incompatible with peritransplant tacrolimus GVHD prophylaxis. Targeting VISTA exclusively on host cells or donor CD8+ T cells was not protective against GVHD lethality. Instead, anti-VISTA mAb–mediated deletion of alloreactive donor T cells depended on targeting a third (non-T) cell type. Further mechanistic studies indicated that donor T cells concurrently exposed to anti-VISTA mAb in vivo but not preincubated in vitro before adoptive T-cell transfer were eliminated via Fc receptor (FcR)-mediated phagocytosis. In a lymphoma challenge model, a graft-versus-lymphoma (GVL) effect was fully retained when anti-human VISTA mAb exclusively targeted donor CD4+ T cells, and was delayed but mostly retained when unseparated donor T cells were infused. In a xenogeneic GVHD model, anti-human VISTA mAb reduced donor T-cell expansion, VISTA T-cell expression levels, and recipient lethality. Together, these data support a novel clinical translational pathway in which acute GVHD lethality can be mitigated without negating the GVL effect.

Article Details

Journal Blood
Volume / Issue Vol. 145, Issue 15
Published April 10, 2025
Pages 1687-1700
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (22)

B

Brent H. Koehn

1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN

E

Elizabeth C. Nowak

2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH

S

Sladjana Skopelja-Gardner

A

Asim Saha

1University of Minnesota, Division of Blood and Marrow Transplantation, Department of Pediatrics, Minneapolis, United States

M

Michael C. Zaiken

1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN

J

Jeremy Allred

1University of Minnesota, Division of Hematology, Oncology and Transplantation, Minneapolis, United States

Y

Yiyung Peng

1Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN

W

Wilson L. Davis

2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH

I

Isabelle Le Mercier

4AvenCell Therapeutics Inc, Watertown, MA

N

Nicholas Schwertner

5Lifordi Immunotherapeutics, Burlington, MA

M

Michael J. Molloy

6ImmuNext Inc, Alton, NH

J

Jay Rothstein

4AvenCell Therapeutics Inc, Watertown, MA

C

Catherine Carriere

4AvenCell Therapeutics Inc, Watertown, MA

M

Megan J. Riddle

1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN

C

Cindy R. Eide

1Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Department of Pediatrics, Masonic Cancer Center, University of Minnesota, Minneapolis, MN

J

Jakub Tolar

1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN

A

Angela Panoskaltsis-Mortari

K

Kyle D. Smith

1Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN

K

Keli L. Hippen

1Division of Pediatric Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota Cancer Center, Minneapolis, MN

T

Tae Kon Kim

1Vanderbilt University Medical Center, Division of Hematology/Oncology, Department of Medicine, Nashville, United States

R

Randolph J. Noelle

2Department of Microbiology and Immunology, Dartmouth Cancer Center, Geisel School of Medicine at Dartmouth, Lebanon, NH

B

Bruce R. Blazar

Department of Pediatrics, Division of Blood & Marrow Transplant & Cellular Therapy, University of Minnesota