Patient-derived xenograft models in musculoskeletal malignancies

Table 3 Selected preclinical studies correlating PDX treatment results with clinical data

Tumor (Refs.)	PDX (n)	Agent	Target	Results	Clinical correlation
OS [33, 54, 56,57,58,59, 61]	1	bortezomib	Proteasome	Combination of bortezomib and adriamycin shows strong TGI ablitily	NA
	2	BHQ880	Wnt signaling	Inhibit tumor growth and metasitasis	NA
	15	IFN-α		Significant TGI in all models, dose dependent	NA
	4	IPI-926	Hedgehog signaling	Significant TGI in 2 of 4 models	NA
	1	Pectolinarigenin	STAT3 signaling	Inhibit tumor growth and metasitasis	NA
EWS [60, 66]	2	WNT974	Porcupine	Delay the early metastasis	NA
	1	SN-38 matrices	Topoisomerase I	Delay the tumor recurrence	NA
SS [63, 65, 67]	1	VX970 [63]	ATR	Significant TGI	NA
	3	tazemetostat	EZH2	Significant TGI in 2 of 3 models	NA
	1	ALGP-DOX	Cytotoxic agents	Significant TVI	NA
SFT [33]	2	DOX, IFO, DTIC, eribulin, trabectedin	Cytotoxic agents	DOX/DTIC, DTIC/IFO, DOX/IFO, eribulin, trabectedin shows strong TVI ablitily	Response to DOX/DTIC in PDXs was concordant with clinical data in 6 out of 12 patients
LPS [64, 67]	2	Pazopanib	Tyrosine kinase	Significant TGI	NA
	2	ALGP-DOX	Cytotoxic agents	Tumor volume stabilisation	NA
RMS [50,51,52, 62]	6	Melphalan	Cytotoxic agents	Produce CR in 5 out of 6 models	10 of 13 untreated patients gain PR after receiving melphalan
	2	Tideglusib	GSK-3β	Negative results	NA
	6	Topotecan, irinotecan	Topoisomerase I	Produce CR in 4 out of 6 models, and CR in 5 out of 6 models, respectively	22 out of 48 patients gain clinical response (CR in 2, PR in 20) after receiving topotecan

Ref reference, n number, GSK-3 β glycogen synthase kinase-3beta, DTIC dacarbazine, DOX doxorubicin, IFO ifosfamide, ADM adriamycin, TGI tumor growth inhibition, TVI tumor volume inhibition, CR complete regression, PR partial regression

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