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Table 2 Clinical trials with CSF1/CSF1R inhibitors in combination with anti-tumor therapies (excluding cancer-immunotherapy doublets)

From: Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy

Class

Target

Compound

Combination partner

Clinical phase

Sponsor

Indication

ClinicalTrials.gov identifier

Status/Results

Reference

Small molecules

CSF1R (and cKIT, Flt3)

Pexidartinib

(PLX3397, PLX108-01)

Androgen-deprivation therapy plus external radiotherapy

1

Plexxikon/Daiichi Sankyo

Prostate cancer

NCT02472275

Ongoing

-

Paclitaxel

1

Plexxikon/Daiichi Sankyo

Solid tumors

NCT01525602

ORR: 4/23 (17%)

CBR: 14/23 (61%)

[35]

Eribulin

1/2

Plexxikon/Daiichi Sankyo

Breast cancer

NCT01596751

Ongoing

-

Temozolomide plus external radiotherapy

1/2

Plexxikon/Daiichi Sankyo

GBM

NCT01790503

Not yet reported

-

Vemurafenib

1

Plexxikon/Daiichi Sankyo

BRAF-mutated melanoma

NCT01826448

Terminated

-

PLX9486 (KIT inhibitor)

1/2

Plexxikon/Daiichi Sankyo

GIST

NCT02401815

Ongoing

-

Sirolimus

1/2

Plexxikon/Daiichi Sankyo

Advanced sarcomas, MPNST

NCT02584647

Ongoing, not yet reported

[73]

Monoclonal antibodies

CSF1R

Emactuzumab (RG7155)

Paclitaxel

1

Roche

Ovarian and breast cancer

NCT01494688

Not yet reported

-

CSF1R

Emactuzumab (RG7155)

Paclitaxel plus bevacizumab

2

Roche

Ovarian cancer

NCT02923739

Ongoing

-

CSF1

MCS110

Carboplatin plus gemcitabine

2

Novartis

TNBC

NCT02435680

Ongoing

-

CSF1

PD-0360324

Cyclophosphamide

2

Pfizer

Recurrent platinum-resistant epithelial ovarian, peritoneal, or fallopian tube cancer

NCT02948101

Ongoing

-

  1. CBR clinical benefit rate, CSF1 colony-stimulating factor 1, CSF1R colony-stimulating factor 1 receptor, dt-GCT diffuse-type tenosynovial giant cell tumor, GBM glioblastoma, GIST gastrointestinal stromal tumor, MPNST malignant peripheral nerve sheath tumor, ORR objective response rate, TNBC triple-negative breast cancer