Wnt inhibition alleviates resistance to anti-PD1 therapy and improves antitumor immunity in glioblastoma

S Shanmugarajan Krishnan (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) S Somin Lee (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) Z Zohreh Amoozgar (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) S Sonu Subudhi A Ashwin Srinivasan Kumar (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) J Jessica M. Posada (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) N Neal Lindeman (Department of Pathology, Brigham and Women’s Hospital) P Pinji Lei (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) M Mark Duquette S Sophie Steinbuch (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) M Marc Charabati P Peigen Huang (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) P Patrik Andersson (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) M Meenal Datta (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) L Lance L. Munn (Edwin L. Steele Laboratories, Department of Radiation Oncology, Massachusetts General Hospital and Harvard Medical School) D Dai Fukumura (Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School) R Rakesh K. Jain

Abstract

Wnt signaling plays a crucial role for many developmental processes. It is also pivotal in the generation and limited treatment outcomes of glioblastoma (GBM). Here, we identified Wnt7b, which is markedly upregulated in GBM patients, as a determinant of resistance to immune checkpoint blockers (αPD1; anti-Programmed Cell Death Protein 1) in a clinically relevant, αPD1-resistant GBM murine model with abundant stem cells. We observed that increased levels of Wnt7b and β-catenin correlated with the resistance to αPD1. Treatment combining a porcupine inhibitor WNT974 with αPD1 reprogrammed the immune suppressive tumor microenvironment (TME) to bolster antitumor immune responses and extended the survival of mice bearing orthotopic GBM, with 25% long-term survivors. Our causal studies revealed that WNT974 potentiated αPD1 therapy by the expansion of antigen presenting DC3-like dendritic cells (DCs). Additionally, WNT974 combination with αPD1 was associated with a reduction in immune suppressive granulocytic myeloid-derived suppressor cells (MDSCs), an increase in the Ki67+CD8/Ki67+regulatory T cells (Treg) ratio, tilting the CD8:Treg balance in the TME toward antitumor immune response, and more pronounced GrzB+CD8+ effector T cells. Conversely, an increase in monocytic MDSCs and phosphorylation of pro-oncogenic proteins was associated with resistance to the combination therapy. Collectively, our preclinical findings provide a strong rationale to test Wnt7b/β-catenin inhibition with αPD1 therapy in GBM patients with elevated Wnt7b/β-catenin signaling.

Article Details

Volume / Issue Vol. 122, Issue 38
Published September 23, 2025
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (17)

S

Shanmugarajan Krishnan

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

S

Somin Lee

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

Z

Zohreh Amoozgar

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

S

Sonu Subudhi

A

Ashwin Srinivasan Kumar

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

J

Jessica M. Posada

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

N

Neal Lindeman

Department of Pathology, Brigham and Women’s Hospital

P

Pinji Lei

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

M

Mark Duquette

S

Sophie Steinbuch

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

M

Marc Charabati

P

Peigen Huang

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

P

Patrik Andersson

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

M

Meenal Datta

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

L

Lance L. Munn

Edwin L. Steele Laboratories, Department of Radiation Oncology, Massachusetts General Hospital and Harvard Medical School

D

Dai Fukumura

Department of Radiation Oncology, Edwin L. Steele Laboratories, Massachusetts General Hospital and Harvard Medical School

R

Rakesh K. Jain