Alternative preclinical models to revolutionize cardiac and CNS drug discovery

Animal models have been vital to understanding disease mechanisms and clinical evaluation of drugs. Despite this, there is mounting evidence showing their lack of physiological relevance and inability to capture the complexity and functional context of human biology. Ethical concerns and high costs of animal research have also limited their applicability in human disease modeling and drug discovery.

Human derived primary tissues and immortalized cell lines help circumvent ethical concerns but are difficult to obtain and limited in availability. Pharmaceutical and biotech industries have been suffering from lack of translationally reliable models. This is reflected in the low rate of success of clinical trials, especially for drugs targeting the central nervous system and cardiac system.

The discovery of induced pluripotent stem cells (iPSCs) wherein somatic cells from the human body could be turned into pluripotent stem cells is a game-changer for science and medicine. Human iPSCs can give rise to multiple cell types and can be derived from any individual, for eg. a patient with a relevant pathology.

The lack of relevant models also affects industry as well as organizations conducting basic and translational research. iPSC derived models can contribute to different stages of the drug discovery pipeline, starting from target identification, lead optimization to pre-clinical trials by:

1. Unraveling human-specific patho-mechanisms

2. Predicting the adverse effects of drugs before market launch

3. Discovering new therapeutic targets with fewer adverse effects

4. Discovering etiology-specific therapies

The webinar will discuss case studies to highlight how the use of iPSC derived models has revolutionized drug discovery. It will also explain how, you, as either a researcher or supporting staff in academia or industry can harness the power of human stem cell technology to your benefit.