This Small Business Innovation Research (SBIR) Phase II project will fund a continuation of breakthrough research, development and commercialization of an in vitro assay to help prevent birth defects. This innovative product is driven by a need to create a test for human developmental toxicity that is more accurate than current tests that use animals. False negative results from these animal assays have lead to unexpected cases of birth defects, such as observed with Thalidomide. This assay, performed on human embryonic stem cells, is more predictive of developmental toxicity than animal models (80% vs 60%) and unlike animal models, provides data about specific human biochemical pathways that are affected. This will fund research to 1) identify biomarkers of developmental toxicity present in three different human cell lines, 2) optimize automation systems, 3) create a web-based interface to be used by customers, 4) standardize and create quality control procedures and 5) take the initial steps required for validation of the assay by the European Centre for the Validation of Alternative Methods (ECVAM). Upon validation, the test will be required in Europe for assessing developmental toxicity of newly developed pharmaceuticals and may be further used for testing of environmental chemicals as well.
The broader impacts of this research include 1) a global reduction in drug and chemical induced human birth defects 2) significant cost savings (up to $70 million per drug) for pharmaceutical companies allowing greater confidence in drug candidate selection and 3) a major global reduction in animal testing.
