Completed Heart, Stroke & Blood Cells, Biochemistry & Physiology

Stem Cells for Safer Medicine

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AI plain-English summary

Drug companies are growing miniature human livers in the lab from stem cells to test whether new drug candidates will poison patients before they ever reach a clinical trial. Liver toxicity is one of the most common reasons promising drugs fail during development, often after years of investment. Current safety tests rely on animal cells or simple cell cultures that do not reliably predict how a human liver will react. This consortium of pharmaceutical companies and academic labs aims to replace those crude screens with living human liver-like cells—called hepatocytes—grown from pluripotent stem cells. The challenge is making those lab-grown cells behave like real adult liver cells, responding to drugs in the same way a patient’s liver would. If the consortium succeeds, drug developers could spot toxic compounds earlier, before expensive animal studies and human trials. That would reduce the number of late-stage drug failures, cut development costs, and—most importantly—prevent patients from being exposed to dangerous side effects. The project is also building automated, high-throughput screening platforms so the tests can be integrated into the standard industrial pipelines that pharmaceutical companies already use to evaluate thousands of compounds.

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Stem Cells for Safer Medicines is a public/private partnership which operates as pre-competitive consortium of industrial and academic partners. The objective of the project is to develop and validate novel in vitro safety screens using pluripotent stem cell lines for early predictive screening which can be scaled up, automated and integrated into current screening technology platforms used in the pharmaceutical industry for the discovery and development of potential new medicines. The project is focussed upon the development of screens for detecting liver toxicity which represents a major cause of attrition during drug development. Effort is currently ongoing to improve the efficiency of the differentiation of pluripotent stem cells to produce hepatocyte-like cells which have the characteristics to replicate the functional activity of adult human hepatocytes and demonstrate the relevant response to drugs in order to provide a reliable and predictive indication of potential toxicity in human patients.

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Collaborative R&D

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