A single dose of a CD137 antibody–drug conjugate protects rhesus macaque allogeneic HCT recipients against acute GVHD

U Ulrike Gerdemann (1Boston Children's Hospital / Dana-Farber Cancer Institute, Hematology/Oncology, Boston, United States) K Kyle Kimler (4Cell Discovery Network of Boston Children's Hospital, Boston Children’s Hospital, Boston, MA) M Matthew R. Warren (1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA) C Connor McGuckin (1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA) R Ryan A. Fleming (1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA) M Matthew R. D’Ambra (6Center for Transplantation Sciences, Department of Surgery, Massachusetts General Hospital, Boston, MA) A Alal Eran (1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA) A Alexandre Albanese (1Boston Children's Hospital, Department of Pediatrics, Boston, United States) E Edward Chen M Marlana Winschel (2Boston Children's Hospital, Division of Hematology/Oncology, Boston, United States) L Lorenzo Cagnin (1Boston Children's Hospital, Department of Pediatrics, Boston, United States) J Jennifer Lane (1Boston Children's Hospital / Dana-Farber Cancer Institute, Hematology/Oncology, Boston, United States) L Lev Gorfinkel (1Boston Children's Hospital, Department of Pediatrics, Boston, United States) B Bartley Adams (7Duke Transplant Center, Department of Surgery, Duke University Medical Center, Durham, NC) J Jean Kwun (7Duke Transplant Center, Department of Surgery, Duke University Medical Center, Durham, NC) L Leanne Lanieri (8Magenta Therapeutics, Cambridge, MA) M Megan Hoban (1Orna Therapeutics, Watertown, United States) T Tahirih L. Lamothe (8Magenta Therapeutics, Cambridge, MA) S Sharon Hyzy (3Vor Bio, Cambridge, United States) L Lisa M. Olson (8Magenta Therapeutics, Cambridge, MA) A Angela Panoskaltsis-Mortari S Susan E. Prockop (1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA) B Bruce R. Blazar (Department of Pediatrics, Division of Blood & Marrow Transplant & Cellular Therapy, University of Minnesota) L Leslie S. Kean (Dana–Farber Cancer Institute–Boston Children’s Hospital, Boston) V Victor Tkachev (5Massachusetts General Hospital, Charlestown, United States)

Abstract

Abstract Rapid CD137 upregulation on alloreactive T cells upon allogeneic stimulation suggests that their selective elimination could prevent acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (HCT). Here, we developed a novel aGVHD prophylactic regimen consisting of a single dose of an anti-CD137 antibody–drug conjugate (CD137-ADC) administered on the day of transplant without additional immunosuppression. The CD137-ADC depleted both human and nonhuman primate (NHP) activated T cells and proved highly effective in preventing xenogeneic aGVHD in mice receiving human peripheral blood mononuclear cells, as well as in NHP undergoing major histocompatibility complex (MHC)-haploidentical HCT. Flow cytometry analysis of NHP T cells indicated specific depletion of activated PD-1+ CD4 and CD8 T cells, while sparing naïve and PD-1−OX40+ memory T-cell subsets during the first week after HCT. CD137-ADC–treated NHP recipients demonstrated robust hematopoietic and immune reconstitution. Hallmarks of T-cell recovery after CD137-ADC, which were associated with long-term aGVHD-free survival, included reconstitution of CD4 memory T cells expressing TRAIL, terminally differentiated CD8 T cells expressing CX3CR1, and CD4 FoxP3+ regulatory T cells, cell types not expected to be involved in aGVHD pathogenesis. CD137-ADC–treated recipients demonstrated a higher risk of reactivation of rhesus lymphocryptovirus (the rhesus macaque Epstein-Barr virus analog), which was associated with reconstitution of follicular helper T cells, interferon signaling–associated memory, and γδT-cell subsets. This reactivation was controllable with rituximab administration. These results document effective depletion of alloreactive T cells and prevention of aGVHD after a single dose of CD137-ADC, suggesting that clinical translation should be carefully explored.

Article Details

Journal Blood
Volume / Issue Vol. 146, Issue 9
Published August 28, 2025
Pages 1127-1141
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (25)

U

Ulrike Gerdemann

1Boston Children's Hospital / Dana-Farber Cancer Institute, Hematology/Oncology, Boston, United States

K

Kyle Kimler

4Cell Discovery Network of Boston Children's Hospital, Boston Children’s Hospital, Boston, MA

M

Matthew R. Warren

1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA

C

Connor McGuckin

1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA

R

Ryan A. Fleming

1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA

M

Matthew R. D’Ambra

6Center for Transplantation Sciences, Department of Surgery, Massachusetts General Hospital, Boston, MA

A

Alal Eran

1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA

A

Alexandre Albanese

1Boston Children's Hospital, Department of Pediatrics, Boston, United States

E

Edward Chen

M

Marlana Winschel

2Boston Children's Hospital, Division of Hematology/Oncology, Boston, United States

L

Lorenzo Cagnin

1Boston Children's Hospital, Department of Pediatrics, Boston, United States

J

Jennifer Lane

1Boston Children's Hospital / Dana-Farber Cancer Institute, Hematology/Oncology, Boston, United States

L

Lev Gorfinkel

1Boston Children's Hospital, Department of Pediatrics, Boston, United States

B

Bartley Adams

7Duke Transplant Center, Department of Surgery, Duke University Medical Center, Durham, NC

J

Jean Kwun

7Duke Transplant Center, Department of Surgery, Duke University Medical Center, Durham, NC

L

Leanne Lanieri

8Magenta Therapeutics, Cambridge, MA

M

Megan Hoban

1Orna Therapeutics, Watertown, United States

T

Tahirih L. Lamothe

8Magenta Therapeutics, Cambridge, MA

S

Sharon Hyzy

3Vor Bio, Cambridge, United States

L

Lisa M. Olson

8Magenta Therapeutics, Cambridge, MA

A

Angela Panoskaltsis-Mortari

S

Susan E. Prockop

1Division of Pediatric Hematology-Oncology, Boston Children’s Hospital, Boston, MA

B

Bruce R. Blazar

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

L

Leslie S. Kean

Dana–Farber Cancer Institute–Boston Children’s Hospital, Boston

V

Victor Tkachev

5Massachusetts General Hospital, Charlestown, United States