Table 1 Latest hiPSCs drug screening
Research field | hiPSCs-derived cells | Drugs tested | Outcome | Ref |
|---|---|---|---|---|
Colon cancer | Cardiomyocytes and endothelial cells, cocultured with tumor spheroids | 2 anticancer drugs were tested in order to simultaneously assess cardiac toxicity and antitumor effects | Demonstration of the feasibility to simultaneously assess cardiac toxicity and antitumor drugs effects | [16] |
Lesch-Nyhan disease (OMIM #300322) | Cortical neurons | Test of 3838 compounds | Identification of 6 pharmacological compounds correcting phosphoribosyltransferase (HGPRT) deficiency–associated neuronal phenotypes | [17] |
Progressive fibrosis | Mesenchymal-like cells | Libraries of ~ 17000 small molecules | Identification of anti-fibrotic small molecule | [18] |
Neuroprotective activity of pharmacological compounds | Embryoid bodies, neuronal precursors and neurons | Screening of peptides of the melanocortin family and endocannabinoids for cytotoxicity, embryotoxicity, and neuroprotective potential | Both melanocortin peptides and endocannabinoids exerted neuroprotective effects | [19] |
Pro-regenerative drug development | Cardiomyocytes | Test of 150 small molecules with pro-regenerative potential | Identification of 2 pro-proliferative compounds acting via the mevalonate pathway. | [20] |
Alzheimer’s disease (OMIM #104300) | Neurons | Test of R33 molecule | R33 is able to reduce Aβ and pTAU | [21] |
Schizophrenia (SZ) | Neural progenitor cells | Screening of 135 drugs with predicted or known interactions in SZ-biology | Identification of 52 drugs ameliorating the SZ-related transcriptomic signature hiPSCs-derived neural progenitor cells | [22] |
Amyotrophic lateral sclerosys (ALS) (OMIM #105400) | Motor neurons | 1416 compounds involved in motor neurons survival | Identification of Src/c-Abl inhibitors increasing the survival rate of ALS motor neurons | [23] |
Homozygous familial hypercholesterolemia (OMIM #144010) | Hepatocytes | Screening of a library of 2320 existing drugs | Identification of 5 cardiac glycosides able to reduce the hepatocytes production of apoB | [24] |
Long-QT syndrome | Cardiomyocytes | Test of LUF7346, known hERG allosteric modulators | LUF7346 is able to rescue some phenotypic features of Long-QT-syndrome | [25] |