EDSP Test Guidelines and Guideline Modifications ELLEN MIHAICH, PH.D., DABT ENVIRONMENTAL AND REGULATORY RESOURCES ISRTP WORKSHOP DECEMBER 13, 2010 1 2 Courtesy of Tim Ward-ABC Laboratories 890 Series In Vitro Screens Tier 1 Assay 3 Guideline Estrogen Receptor (ER) Binding Assay USEPA 890.1250 Estrogen Receptor Transcriptional Activation Assay USEPA 890.1300 OECD 455 Androgen Receptor (AR) Binding Assay USEPA 890.1150 Aromatase Assay USEPA 890.1200 Steroidogenesis Assay USEPA 890.1550 Purpose An ER binding assay that utilizes rat uterine cytosol to examine the ability of a test chemical to bind with estrogen receptors An second type of ER binding assay that uses a human cell line to examine the ability of a test chemical to bind with estrogen receptors and alter gene transcription An AR binding assay that utilizes rat prostate cytosol to examine the ability of a test chemical to bind with androgen receptors Aromatase is an enzyme complex responsible for estrogen biosynthesis that converts androgens into estrogens, estradiol, and estrone. The Aromatase in vitro assay uses a human recombinant form of the protein and focuses on this portion of the steroidogenic pathway to detect substances that inhibit aromatase activity. The Steroidogenesis in vitro assay utilizes the H295R human adrenocortical carcinoma cell line to detect interference with the body’s production of male and female steroid sex hormones (estrogen and testosterone). 890 Series In Vivo Screens Tier 1 Assay 4 Guideline Uterotrophic Assay USEPA 890.1600 OECD 440 Hershberger Assay USEPA 890.1400 OECD 441 Male Pubertal Assay USEPA 890.1500 Female Pubertal Assay USEPA 890.1450 Fish Short-Term Reproduction Assay USEPA 890.1350 OECD 229 Amphibian Metamorphosis Assay USEPA 890.1100 OECD 231 Purpose Ovariectomised or immature female rats are used to screen for estrogenic effects by measuring uterine weight changes. Designed to detect chemicals that are androgenic, antiandrogenic or inhibit 5α-reductase. Accessory sex gland weights, including several androgen-dependent tissues, are measured in castrated or immature male rats Androgenic, anti-androgenic, and thyroid activity is screened in male rats during sexual maturation. Abnormalities associated with sex organs and puberty markers, as well as thyroid tissue are examined. Estrogenic and thyroid activity is screened in female rats during sexual maturation. This assay examines abnormalities associated with sex organs and puberty markers, as well as thyroid tissue. Screens for estrogenic and androgenic effects. The assay examines abnormalities associated with survival, reproductive behavior, secondary sex characteristics, histopathology, and fecundity (i.e., number of spawns, number of eggs/spawn, fertility, and development of offspring) of fish exposed to test chemicals. Involves the use of tadpoles to determine if chemicals affect the thyroid during metamorphosis and consequently result in developmental effects. Test Order Restriction Requirement to perform screens according to 890 series guideline “…you may not deviate from an approved testing protocol unless you first consult with the Agency and obtain Agency approval of any planned deviation.” 5 General and Specific Guideline Issues • Primarily hitting the high points of some of the screens today • For more information please see the Test Guideline comments sent by the EPF in the EPA docket: EPA-HQ-OPP-2009-0634-0135 6 General Guideline Issues *No public review of guidelines prior to publication • Very prescriptive and inflexible • Test validity criteria too stringent • Sensitivity and specificity issues • Typographical errors • No standard evaluation procedures • No defined Weight of Evidence Procedures for the battery 7 ER and AR Binding Assays Shortcomings No metabolic capability Impacted by pH, denaturation, precipitates and particulates Cannot distinguish agonists/antagonists Validation and Guideline Issues Inconsistency in rat uterine cytosol preparation Inconsistency in cytosolic prostate gland preparation Numerous typographical errors 8 ER Transcriptional Activation Shortcomings No metabolic capability Validation and Guideline Issues Only validated for ER agonists Limited validation and false positive rate high EPA’s recommendation for positive response (PC10) deviates from peer review recommendation EPA’s acceptable lower level of dynamic range of 4fold induction raises issue of distinguishing from background noise 9 Aromatase (Human Recombinant) Shortcomings No metabolic capability Impacted by denaturation of protein Validation and Guideline Issues Limited validation Numerous typographical errors 10 Steroidogenesis (Human Cell Line) • Shortcomings Cytotoxicity confounds results Presence of any detergent residue on glassware can confound assay results • Validation and Guideline Issues High variability between laboratories during validation make the results difficult to interpret The guideline is poorly organized with no stated purpose – more of a protocol than guidance Very small pipetting volumes could lead to errors Parallel evaluation of cytotoxicity needed. Guideline permits up to 20% cytotoxicity while ICCVAM recommended only 10% 11 Uterotrophic • Shortcomings Route of administration and animal model compared to relevance • Validation and Guideline Issues While there are references to the anti-estrogenic component of the study, there has been no validation of this and anti-estrogenicity should not be assessed Dose route and animal model preferences not harmonized with OECD - environmental relevance and metabolism should be considered 12 Hershberger • Validation and Guideline Issues 13 Wide inter-laboratory variation in the mean age at which preputial separation occurs Clarity needed for the interpretation of study with and without optional endpoints EPA recommends use of multivariate analyses of all accessory sex organ weights in cases where only a single tissue gives a significant response; such posthoc analyses are useful for hypothesis generation, but should not be used in hypothesis testing Anesthetic agent and euthanizing method should be chosen carefully to avoid artifacts if performing optional steroid measurement CV’s for control and high dose organ weights should be reported. Deviation of more than 3 could result in study rejection Pubertals • Shortcomings High sensitivity but low specificity Apical endpoints provide only limited information on the mode of action for potential endocrine-active chemicals Significant inherent biological variability in the endpoints (puberty onset, estrous cycle, organ weights) complicates interpretation • Validation and Guideline Issues Validation studies did not demonstrate a negative response using a true negative control agent Dose selection is critical to avoid non-specific outcomes 14 Amphibian Metamorphosis • Shortcomings Apical endpoints with unknown specificity Poorly soluble or unstable compounds difficult to test Not a short screen • Validation and Guideline Issues No known negative compounds Dose setting guidance needed Background levels of iodide in food and water may make comparison between labs difficult Developmental staging 15 Fish Short-Term Reproduction • Shortcomings Multiple modes of action detected – apical endpoints Medium sensitivity, low specificity Long and expensive “screen” • Validation and Guideline Issues High variability observed in plasma sex steroids suggests they are not particularly robust endpoints Statistical power of the fecundity endpoint is low Limited quantities of blood plasma may require prioritization of most robust measurements, such as vitellogenin, rather than sex steroids Dose setting is critical to avoid confusing systemic toxicity with genuine endocrine-mediated effects 16 Sensitivity and Specificity The Series 890.1350 TG (fish assay) states: "It is recognized that some endpoints may be responsive to nonendocrine stresses in addition to endocrine-mediated pathways, particularly fecundity. Although reductions in fecundity indicate adverse organismal and, potentially, population level effects (i.e., reproductive toxicity), these cannot be definitively distinguished from direct endocrinemediated effects by this assay when changes in other core endpoints are not present. Nevertheless, reductions in fecundity are considered a positive effect in this assay because they may be endocrine-mediated ..." 17 Staging the Conduct of the Screens Stage 1 Stage 2 Stage 3 Stage 4 18 •Estrogen Receptor (ER) Binding •Androgen Receptor Binding •ER Transcriptional Activation •Uterotrophic Assay •Hershberger Assay •Aromatase Assay •Steroidogenesis Assay •Male Pubertal Assay •Female Pubertal Assay •Fish Short-Term Reproduction Assay •Amphibian Metamorphosis Assay Summary • The current US EPA EDSP falls short in meeting many of the attributes of an efficient and effective screening program. 19 Many of the assays are not mechanistic, and some have yet to be shown to meet the basic requirement of distinguishing an endocrine active substance from a negative control or differentiating potential endocrine-mediated responses from responses via other modes of action (e.g. hepatotoxicity) or systemic toxicity. The prescriptive nature of the guidelines combined with typographical errors, overly conservative validity criteria, and inflexible test order requirements likely mandate protocol approval prior to conducting the tier 1 battery Summary-cont. • A staged approach to the performance of the screens may improve the interpretability of the results, increasing the efficiency of the work and clarity of the results. Efficiency and clarity are essential because interpretation of the entire battery is the determinant for proceeding to Tier 2. 20 • While staging the EDSP ESB may improve interpretation as to whether a substance may interact with components of the estrogen, androgen, and thyroid hormone systems, such activities cannot overcome inherent limitations of the tier 1 screens. • EPA EDSP ESB Test Guidelines can be accessed at http://www.epa.gov/ocspp/pubs/frs/publications /Test_Guidelines/series890.htm 21