Table 1.

Correlation between high‐throughput drug screening (HTS) drug hits and prior molecular analysis.

Patient ID	Diagnosis	Drug hit	Drug target	Molecular target	HTS correlated with molecular
RA‐002	HGG	Everolimus	mTOR	TSC1 mutation with LOH	Yes
		Sirolimus	mTOR		Yes
		Temsirolimus	mTOR		Yes
		Sorafenib	Multi TKI	BRAF 6 copies and high RNA	Yes
RA‐028	HGG	Crenolanib	Multi TKI	PDGFRA mutation	Yes
		Ponatinib	Multi TKI		Yes
RA‐056	HGG	Dasatinib	Multi TKI	PDGFRA mutation	Yes
		Pazopanib	Multi TKI		Yes
RA‐034	CPC	Dasatinib	Multi TKI	High SRC RNA	Yes
RA‐055	DMG	Nintedanib	Multi TKI	PDGFRA and VEGFR2 amp	Yes
RA‐019	EWS	Cabozantinib	Multi TKI	High KIT RNA	Yes
WE‐012	EWS	Gefitinib	EGFR	EGFR 6 copies and high RNA	Yes
RA‐017	OST	Dinaciclib	CDK1/2/5/9	High CCNE1 RNA	Yes
		PRI‐724	CTNNB1	High CTNNB1 RNA	Yes
		Panobinostat	HDAC	High HDAC6 RNA	Yes
RA‐013	NBL	Ceritinib	ALK, IGF1R	High ALK and IGF1R RNA	Yes
		Venetoclax	BCL2	High BCL2 RNA	Yes
WE‐005	OST	Crizotinib	ALK, MET, ROS1	NA
		Temsirolimus	mTOR
RA‐002	HGG	Axitinib	multi TKI	HTS drug responses without prior molecular hallmarks for sensitivity to that drug
		Lapatinib	ERBB2, EGFR
		Vandetanib	multi TKI
RA‐028	HGG	Lapatinib	ERBB2, EGFR
RA‐056	HGG	Abiraterone	CYP17A1
		Fulvestrant	ESR1
		Pinometostat	DOT1L
RA‐019	EWS	Alectinib	ALK
		Dinaciclib	CDK1/2/5/9
		Panobinostat	HDAC
RA‐054	RMS	Buparlisib	PI3K
		Voxtalisib	PI3K, mTOR
RA‐017	OST	Crenolanib	multi TKI

Of the 17 HTS performed, 32 molecular drug hits were identified in 11 samples.

amp, amplification; CPC, choroid plexus carcinoma; DMG, diffuse midline glioma H3 K27M mutant; EWS, Ewing’s sarcoma; HGG, high grade glioma; LOH, loss of heterozygosity; TKI, tyrosine kinase inhibitor; NA, molecular data not available; NBL, neuroblastoma; OST, osteosarcoma; RMS, rhabdomyosarcoma.