Table 1 FDA-approved drugs and clinical trials targeting CSCs

From: Cancer stem cells: landscape, challenges and emerging therapeutic innovations

Drug/Intervention

Target/Mechanism

Cancer Type

ClinicalTrial ID

Phase

vismodegib

Hedgehog inhibitor

basal cell carcinoma

NCT02436408

FDA-approved

metformin

OXPHOS inhibitor

ovarian cancer

NCT01579812

Phase II

doxycycline

Mitochondrial biogenesis inhibitor

pancreatic cancer

NCT02775695

Phase II

defactinib

FAK inhibitor

non-small cell lung cancer

NCT01951690

Phase II

reparixin

CXCR1 inhibitor

triple-negative breast cancer

NCT02370238

Phase II

HER2-negative metastatic breast cancer

NCT02001974

Phase I

MK0752

Gamma-secretase inhibitor (Notch)

advanced/metastatic breast cancer

NCT00645333

Phase I/II

IPI-926

Hedgehog inhibitor

head and neck cancer

NCT01255800

Phase I

fursultiamine

ABCB1 and ABCG2 transporter inhibitor

esophageal squamous cell carcinoma

NCT02423811

Phase II

imetelstat

Telomerase inhibitor

non-small cell lung cancer

NCT01137968

Phase II

CSC vaccine

Cancer stem cell vaccine

nasopharyngeal cancer

NCT02115958

Phase I/II

hepatocellular carcinoma

NCT02089919

Phase I/II

lung cancer

NCT02084823

Phase I/II

ovarian cancer

NCT02178670

Phase I/II

colorectal cancer

NCT02176746

Phase I/II

pancreatic cancer

NCT02074046

Phase I/II

dendritic cell therapy

CSC-targeting immunotherapy

glioblastoma

NCT03548571

Phase II/III

CSC antigen-targeted vaccine

glioblastoma

NCT02010606

Phase I

STEMVAC

CD105/Yb-1/SOX2/CDH3/MDM2-polyepitope DNA vaccine

triple-negative breast cancer

NCT05455658

Phase II

vismodegib + gemcitabine

Hedgehog inhibitor + chemotherapy

pancreatic cancer

NCT01195415

Phase II

ChemoID-guided Therapy

Chemotherapy guided by CSC

recurrent glioblastoma

NCT03632135

Phase III

bevacizumab

Anti-VEGF therapy with anti-CSC effects

breast cancer

NCT01190345

Phase II

temsirolimus + liposomal doxorubicin

mTOR inhibitor + chemotherapy

sarcoma

NCT00949325

Phase I/II