Clinical trials with CSF1R inhibitors for the treatment of dt-GCT

ClassTargetCompoundClinical identifierStatus/ ResultsReference
Small moleculesCSF1R (and cKIT, Flt3)Pexidartinib (PLX3397, PLX108-01)1PlexxikonSolid tumors and extension for MEC, dt-GCT, GIST, ATC, metastatic solid tumorsNCT01004861OngoingORR: 12/23 (52%)CBR: 19/23 (83%)[22]
3Plexxikon/Daiichi Sankyodt-GCT or GCT-TSNCT02371369Ongoing-
Monoclonal antibodiesCSF1REmactuzumab (RG7155)1RocheSolid tumors and dt-GCTNCT01494688ORR: 24/28 (86%)CBR: 27/28 (96%[12]
CSF1RCabiralizumab (FPA008)1/2FivePrimedt-GCTNCT02471716Ongoing-
CSF1MCS1102Novartisdt-GCT or GCT-TSNCT01643850OngoingORR: 4/4 (100%)[21]

ATC anaplastic thyroid cancer, CBR clinical benefit rate, CSF1 colony-stimulating factor 1, CSF1R colony-stimulating factor 1 receptor, dt-GCT diffuse-type tenosynovial giant cell tumor, GCT-TS giant cell tumor of the tendon sheath, GIST gastrointestinal stromal tumor, MEC mucoepidermoid carcinoma of the lung, ORR objective response rate