Table 1.

Summary of Compounds Identified from Stem Cell-Based Screens

Compound	Starting Population	Screening Readout	Biological Process Affected	Mechanism of Action	References
Cellular Reprogramming
Reversine	C2C12 cells	alkaline phosphatase as an osteoblast marker	reprogramming from lineage-committed myoblasts to multipotent stage	a dual inhibitor of MEK1 and nonmuscle myosin II heavy chain	Chen et al. (2004)
BIX-01294	Oct4, Klf4-transduced mouse embryonic fibroblasts (MEFs)	ESC colony and staining with alkaline phosphatase as a pluripotency marker	reprogramming of Oct4/Klf4-transduced mouse embryonic fibroblasts	a potential G9a histone methyltransferase inhibitor	Shi et al. (2008)
BayK8644				a potential L-channel calcium agonist
Valproic acid	Oct4-, Sox2-, Klf4-, and c-Myc-transduced MEFs	the percentage of Oct4-GFP+ cells	improve reprogramming efficiency of mouse and human fibroblasts	a potential histone deacetylase inhibitor	Huangfu et al., 2008a, Huangfu et al., 2008b
Kenpaullone	Oct4-, Sox2-, and c-Myc-transduced MEFs	Nanog-Luc activity	induce reprogramming in the absence of Klf4	a potential GSK-3β inhibitor	Lyssiotis et al. (2009)
RepSox	Oct4-, Klf4-, and c-Myc-transduced MEFs	the number of Oct4-GFP + colonies	induce reprogramming in the absence of Sox2	inhibit TGF-β signaling	Ichida et al. (2009)
Forskolin	Sox2-, Klf4-, and c-Myc-transduced MEFs	the number of Oct4-GFP + colonies	induce reprogramming in the absence of Oct4	a potential cAMP activator	Hou et al. (2013)
(S)-(+)-Dimethindene maleate	primed H9 hESCs	activate the OCT4 distal enhancer	support the conversion and maintenance of dome-shaped hPSCs from primed hPSCs	a potential M2 muscarinic receptor antagonist	Yang et al. (2017)
minocycline hydrochloride				a potential metalloproteinase inhibitor
Directed Differentiation
PD407824	C2C12 cells	Id2-Luc reporter	synergize with BMP4 to direct hPSC differentiation toward mesoderm or cytotrophoblast stem cells	inhibit checkpoint kinase 1	Feng et al. (2016)
Atauprimide	mESCs	immunostaining using SOX17 antibody	prime mouse and human ESC differentiation	interact with NME2 and inhibit nuclear localization	Zhu et al. (2009)
IDE1	mESC	Sox17 promoter-driven tdTomato	induce nearly 80% of ESCs to form definitive endoderm	activate TGF-β signaling	Borowiak et al. (2009)
IDE2
Indolactam V	hESC-derived endodermal cells	immunostaining using PDX1 antibody	induce differentiation toward pancreatic progenitors	activate PKC	Chen et al. (2009)
H1152	hESC-derived pancreatic progenitors	immunostaining using insulin antibody	increase the generation of insulin-secreting cells	inhibit ROCKII	Ghazizadeh et al. (2017)
CI-994	hPSC-derived immature hepatocytes	albumin-Venus reporter	promote hPSC differentiation toward hepatocyte-like cells	inhibit histone deacetylase	Li et al. (2018)
SU5402 CHIR99021 DAPT	hPSC-derived neuroectoderm	immunostaining using PAX6 and β3-tubulin antibodies	promote hPSC differentiation toward neurons	a potential VEGFR inhibitor	Chambers et al. (2012)
				a potential GSK-3β inhibitor
				a potential Notch inhibitor
Selamectin	mESC	immunostaining using tyrosine hydroxylase antibody	transform mPSCs into neurons	a potential GABAA agonist	Sun et al. (2013)
Phenanthroline	hESC-derived lens placode	SIX1 H2B::GFP reporter	promote differentiation from nonneural ectoderm to cranial placode	a potential metalloprotease inhibitor	Tchieu et al. (2017)
KY02111	monkey ESCs	αMHC promoter-driven EGFP	PSC differentiation toward cardiomyocytes	NA	Minami et al. (2012)
Sodium nitroprusside	mESC-derived mesodermal cells	CCS:lacz reporter	promote the generation of cardiac Purkinje fiber-like cells	increase intracellular cAMP	Tsai et al. (2015)
Stem Cell-Based Disease Models
SKF-86466	wild-type and familial dysautonomia iPSC-derived neural crest cells	alamarBlue to detect cell survival qRT-PCR to detect IKBKAP expression	rescue IKAP protein expression and the disease-specific loss of autonomic neuronal marker expression	a potential α2-adrenoceptor antagonist	Lee et al. (2012)
Kenpaullone	wild-type and SOD1G93A HB9::GFP mESC-derived motor neuron	imaging-based assay to count GFP+ cell number	prolong the healthy survival both mouse and human motor neurons	a potential dual inhibitor of GSK-3 and HGK kinases	Yang et al. (2013)
Alsterpaullone	fibroblasts from parental SMA carriers	immunostaining using SMN antibody	increase cellular SMN	a potential GSK-3 inhibitor	Makhortova et al. (2011)
E34	a co-culture of mESC-derived motor neurons, astrocytes, and interferon-γ- and lipopolysaccharide-activated microglial cells	the total neurite length of all neurites of the motor neurons	prevent the neurons from cytokine and lipopolysaccharide-induced degeneration	inhibit nitric oxide synthase	Hoing et al. (2012)
Pepstatin A	EDNRB-null mutant hESC-derived enteric nervous system precursors	cell migration assay	rescue loss of EDNRB-induced cell migration defects	inhibit BACE2	Fattahi et al. (2016)
Avermectins	TS21 iPSC-derived cortical neurons	the activity of lactate dehydrogenase in medium	increase the relative production of short Aβ peptides at the expense of longer, potentially more toxic peptides	NA	Brownjohn et al. (2017)
Gardiac glycosides	familial hypercholesterolemia iPSC-derived hepatocyte-like cells	ELISA-based assay to detect apolipoprotein B (apoB) production	reduce the production of apoB	potential inhibitors of the membrane sodium-potassium (NA+-K+) pump	Cayo et al. (2017)
T5524	CDKAL1−/− hESC-derived pancreatic β-like cells	staining with PI and insulin antibody	rescues loss of CDKAL1 induced increased sensitivity to glucolipotoxcity	inhibit the FOS/JUN pathway	Zeng et al. (2016)
Galunisertib	GLIS3−/− hESC-derived pancreatic β-like cells	staining with PI and insulin antibody	rescues loss of CDKAL1 induced pancreatic β cell death	inhibit TGF-β pathway	Amin et al. (2018)
Emricasan	hNPCs	caspase-3/7 activity assay	block ZIKV-induced hNPC death	a pan-caspase inhibitor	Xu et al. (2016)
Niclosamide			inhibits ZIKV replication	NA
Hippeastrine hydrobromide	hNPCs	staining with ZIKV antibody	eliminate ZIKV infection	NA	Zhou et al. (2017)
Geneticin	familial adenomatous polyposis-iPSC-derived colonic organoids	staining with CDX2 and Ki67 antibodies	rescue early stop codon caused APC protein decrease and increased cell proliferation	NA	Crespo et al. (2017)

NA, not applicable.