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Updated: May 23, 2025

Human Pluripotent Stem Cell Based Developmental Toxicity Assays for Chemical Safety Screening and Systems Biology Data Generation
Published on: June 17, 2015
NAM-based analysis of contaminant short-term organ toxicity in HepaRG and RPTEC/TERT1 cells
Kristina Jochum1, Andrea Miccoli2, Cornelia Sommersdorf3
1German Federal Institute for Risk Assessment, Department of Pesticides Safety, Berlin, Germany.
Abstract:
New Approach Methodologies (NAMs), including cell culture and multi-level omics analyses, are promising alternatives to animal testing. To become useable for risk assessment purposes, they have to be applicable for different substance groups. One important group of substances is food contaminants, including synthetic chemicals, such as perfluorooctanesulfonic acid (PFOS) and perfluorooctanoic acid (PFOA), and natural compounds, such as mycotoxins and pyrrolizidine alkaloids. We tested five known contaminants affecting the liver and/or the kidney - PFOS, PFOA, Aflatoxin B1 (AB1), lasiocarpine (Las), and cadmium chloride - using HepaRG and RPTEC/TERT1 cells at non-cytotoxic concentrations for 36 and 72 h. Our NAM-based testing protocol included marker protein analysis for cellular functions and targeted transcriptomics followed by bioinformatics pathway analysis. The effects observed were compared with established in vivo results. Protein analysis indicated various affected pathways in HepaRG cells, with generally fewer effects in RPTEC/TERT1 cells. The strongest transcriptional impact was noted for Las in HepaRG cells. This study demonstrated the test protocol's applicability across different substances, revealing significant differences between HepaRG and RPTEC/TERT1 cell lines. RPTEC/TERT1 cells, while expressing renal-specific CYP enzymes, were less suitable for detecting effects of substances requiring hepatic metabolic activation, like Las and AB1. Our data supports the concept of specific pathway toxicity, with pathway analysis enabling the prediction of effects based on mechanism rather than target organ. Employing multiple omics techniques provided comprehensive insights into various compound effects, including steatosis, reactive oxygen species production and DNA damage, highlighting the potential of an extended omics approach for advancing toxicological assessments.
Insights
New Approach Methodologies (NAMs) show promise for replacing animal testing in food contaminant risk assessment. This study validated a NAM protocol using omics and cell cultures, demonstrating its applicability across diverse chemical and natural toxins.
Area of Science:
- Toxicology
- In vitro toxicology
- Omics technologies
Background:
- New Approach Methodologies (NAMs) offer alternatives to animal testing for chemical risk assessment.
- Applicability of NAMs across diverse substance classes, including food contaminants, is crucial for regulatory acceptance.
- Food contaminants encompass synthetic chemicals (e.g., PFOS, PFOA) and natural toxins (e.g., mycotoxins, pyrrolizidine alkaloids).
Purpose of the Study:
- To evaluate the applicability of a NAM-based testing protocol for assessing the toxicity of various food contaminants.
- To compare the responses of HepaRG (liver) and RPTEC/TERT1 (kidney) cell lines to different contaminants.
- To investigate the utility of omics analyses and pathway analysis for predicting toxicological effects.
Main Methods:
- Utilized HepaRG and RPTEC/TERT1 cell lines exposed to five known contaminants (PFOS, PFOA, Aflatoxin B1, lasiocarpine, cadmium chloride) at non-cytotoxic concentrations.
- Employed a NAM protocol including marker protein analysis for cellular functions and targeted transcriptomics.
- Conducted bioinformatics pathway analysis to interpret omics data and compared results with in vivo data.
Main Results:
- Protein analysis revealed pathway alterations in HepaRG cells, with fewer effects observed in RPTEC/TERT1 cells.
- Lasiocarpine exhibited the strongest transcriptional impact in HepaRG cells.
- Significant differences in cell line responses were noted; RPTEC/TERT1 cells were less suitable for detecting hepatically metabolized toxins like lasiocarpine and Aflatoxin B1.
Conclusions:
- The validated NAM protocol is applicable across different substance classes, supporting its use in risk assessment.
- Pathway analysis enables mechanism-based toxicity prediction, independent of the target organ.
- Integrated omics approaches provide comprehensive insights into compound-induced effects, advancing toxicological assessments.

