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CSF1R Inhibition with Chemotherapy Relieves Systemic Immune Suppression in Patients with Metastatic Triple-Negative Breast Cancer and Boosts anti-PD-1 Efficacy in Transgenic Mammary Tumors.

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Clinical cancer research : an official journal of the American Association for Cancer Research 📖 저널 OA 55.2% 2022: 3/4 OA 2023: 6/8 OA 2024: 8/14 OA 2025: 57/92 OA 2026: 84/165 OA 2022~2026 2026
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Poissonnier A, Rugo HS, Horton W, Kim T, DeLuca A, Egeland EV

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[PURPOSE] Significant correlations exist between presence of intratumoral macrophages, tumor progression, and poor outcomes in triple-negative breast cancer (TNBC) with limited therapeutic options ava

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APA Poissonnier A, Rugo HS, et al. (2026). CSF1R Inhibition with Chemotherapy Relieves Systemic Immune Suppression in Patients with Metastatic Triple-Negative Breast Cancer and Boosts anti-PD-1 Efficacy in Transgenic Mammary Tumors.. Clinical cancer research : an official journal of the American Association for Cancer Research. https://doi.org/10.1158/1078-0432.CCR-25-4236
MLA Poissonnier A, et al.. "CSF1R Inhibition with Chemotherapy Relieves Systemic Immune Suppression in Patients with Metastatic Triple-Negative Breast Cancer and Boosts anti-PD-1 Efficacy in Transgenic Mammary Tumors.." Clinical cancer research : an official journal of the American Association for Cancer Research, 2026.
PMID 41894563 ↗

Abstract

[PURPOSE] Significant correlations exist between presence of intratumoral macrophages, tumor progression, and poor outcomes in triple-negative breast cancer (TNBC) with limited therapeutic options available for advanced stage disease. Preclinical studies revealed that inhibition of myelomonocytic colony stimulating factor 1 (CSF1) or its receptor (CSF1R), plus cytotoxic chemotherapy decreased primary tumor growth kinetics and pulmonary metastases by CD8+ T cell-dependent mechanisms. This translational study evaluated CSF1R inhibition combined with eribulin in metastatic TNBC and explored rational preclinical combination strategies with PD-1/PD-L1-blockade based on clinical immune correlate analyses.

[EXPERIMENTAL DESIGN] A nonrandomized, open-label phase 1b/2 trial (NCT01596751) evaluated pexidartinib (PLX3397, PLX), a CSF1R inhibitor (CSF1Ri), plus eribulin mesylate in heavily pretreated individuals with metastatic TNBC. Clinical efficacy was assessed alongside peripheral blood correlates. Preclinical studies in transgenic mammary adenocarcinoma models examined biomarker-driven therapy combinations.

[RESULTS] The 12-week progression-free survival rate was 36% (95% CI, 22.2%-58.4%), with 44.8% of patients achieving clinical benefit; a subset experienced disease control beyond 6-months. Patients with partial response or stable disease demonstrated increased baseline leukocyte activation, including enrichment of CD8+ and CD4+ memory T cells and increased PD-1 expression on CD4+ T cells. In preclinical studies, CSF1Ri expanded the therapeutic index of PD-1 blockade, yielding transient tumor regression in ~60% of mice and a transient expansion of effector and resident memory T cells.

[CONCLUSIONS] These clinical and preclinical findings provide rationale for therapies to increase therapeutic index of aPD-1 therapy by diminishing presence of T cell-suppressive myelomonocytic cells to improve outcomes for patients with refractory disease.