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Diverse cohort, in vitro, for discovery and trial design

The ability to represent a physiologically relevant, diverse cohort, in an in vitro format would provide a powerful platform for drug development and clinical trial design, especially for complex diseases.

A “clinical trial in a dish” refers to a type of laboratory research in which scientists use stem cells or organoids (miniature, lab-grown versions of organs) to model the progression of a disease and test potential treatments. This approach would allow for more efficient and accurate testing of drugs and therapies before they are tested in human clinical trials. Additionally, this type of research can provide valuable insights into the underlying mechanisms of disease.

Statistically relevant power

We see a future where the use of statistically relevant, cohort models are developed which reflect diversity of populations, as a powerful tool for drug discovery and design.

This includes pre-clinical discovery stages where efficacy could be challenges across a diverse number of donor cell co-cultures but even more powerfully, linked to patient selection and patient stratification in the early clinical stages. In this function, the clinical trial in a dish model has the potential to guide a trial to success versus ambiguity.

Cardiotoxicity in a dish

In one review of the application of ‘clinical trial in a dish’, Lam et al. describes the use patient-derived induced pluripotent stem cells to identify risks of drug-induced cardiotoxicity. Drug-induced cardiotoxicity is a significant clinical issue, with many marketed drugs being labeled with warnings on cardiovascular adverse effects. Treatments are often prematurely halted when cardiotoxicity is observed, which limits their therapeutic potential. The review explores the current use of patient-specific induced pluripotent stem cells in identifying populations who are at risk to drug-induced cardiotoxicity and their potential applications in future precision medicine practice. Concluding that induced pluripotent stem cell platforms have the potential to realize personalized medicine.

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