TABLE 1.

Therapies targeting MDS-SCs in clinical trials.

Target	Disease Association	Drug name	Drug type	Intervention	Disease treated	Trial phase identifier
CD123	Overexpressed in HR MDS and AML	SL-401	ADC	SL-401 +	HR MDS and AML	Phase I/II: NCT03113643, NCT00397579
				HMAs +
				Venetoclax
		IMGN632	ADC	IMGN632	MPN, AML, BPDC, ALL	Phase I/II: NCT03386513
		MB-102	CAR-T	IMGN632 +	MPN, AML, BPDCN,ALL	Phase I: NCT02159495
				Cyclophosphamide +
				Fludarabine + Phosphate
TIM3	Overexpressed in HR MDS and AML	MBG453	Blocking antibody	MBG453 +	HR MDS and AML	Phase III: NCT04266301, Phase II: NCT03946670
				HMAs
CD47	Overexpressed in HR MDS and AML	Magrolimab	Blocking antibody	Magrolimab +	HR MDS and AML	Phase III: NCT04313881
				Azacitidine		Phase I: NCT03248479
TLR2	Overexpressed in LR MDS	Tomaralimab	Blocking antibody	Tomaralimab	LR MDS	Phase I/II: NCT02363491, NCT03337451
CXCR2	Overexpressed in MDS	SX-682	Small molecular inhibitor	SX-682	HR MDS	Phase I: NCT04245397
IRAK1	Overexpressed in MDS	Pacritinib	Small molecular inhibitor	Pacritinib +	LR MDS	Phase I: NCT02469415, NCT02564536
				Azacitidine
IRAK4	Overexpressed in MDS	CA-4948	Small molecular inhibitor	CA-4948	HR MDS and AML	Phase I: NCT04278768
STAT3	Overexpressed in MDS	Pyrimethamine	Small molecular inhibitor	Pyrimethamine	HR and IR MDS	Phase I: NCT03057990
SF3B1	Frequently mutated in MDS	H3B-8800	Small molecular inhibitor	H3B-8800	MDS and AML	Phase I: NCT02841540

Abbreviations: MDS, myelodysplastic syndrome; AML, acute myeloid leukemia; BPDCN, Blastic plasmacytoid dendritic cell neoplasm; MPN, myeloproliferative neoplasm; ALL, acute lymphoblastic leukemia; ADC, Antibody-drug conjugate; CAR-T, Chimeric antigen receptor T cell therapy; HR, High-risk; LR, Low-risk.