StemBANCC

There is an alarming failure to deliver new medicinal entities (NMEs) despite large investments by the pharmaceutical industry and by the public sector. Perhaps the most likely explanation for this trend is that current drug discovery programmes are based upon biological models that fail to reflect the patient population, e.g. animal models and inadequate cell culture systems. Whilst very promising chemicals highly specific for a relevant target can be identified within such model systems, they frequently fail when taken out into clinical settings through poor efficacy or unexpected toxicity. Drug discovery based upon human models representative of the patient population would logically represent a great improvement. However it is generally not possible to have access to relevant tissue for high throughput experimentation for many disorders. This then is the great hope for stem cell based disease modeling whereby tissue from a patient can be reprogrammed to pluripotency and then differentiated into a cell type closely resembling the tissue bearing the brunt of disease. Modelling disease with human induced pluripotent stem cell (iPSC) lines in a dish offers many opportunities but for it to be included into a drug-screening platform, there are still many challenges.

The StemBANCC consortium is an academic-industry partnership tasked with addressing these challenges by advancing science and implementing novel, cutting edge technologies. It will establish the framework to provide well characterized patient biomaterials in an accessible and sustainable bio-bank and also demonstrate proof of concept for the utility of the cell culture systems derived from said biomaterial. The principal objective of StemBANCC over 5 years is to fully adopt stem cell technology for drug discovery and to overcome current bottlenecks. There is a need to (1) Develop nonintegrating technologies to rapidly reprogram human cells to pluripotency whilst minimizing non-disease related variability; (2) Differentiate iPSC colonies consistently with greater functional resemblance to the native target cell of interest; (3) Identify disease relevant cellular defects amenable for assay miniaturization; (4) Scale and enhance systemisation of the entire pipeline sufficiently to enable high through-put processing.

The tasks of the NMI are to develop assays for higher throughput analysis of drug on and off effects on the function of stem cell-derived cell types such as cardiomyocytes, neurones and beta cells by means of microelectrode array and automated patch clamp technologies.