Neurite extension from motor neurons to myotubes and synapse formation detected by Bungarotoxin staining

Five key takeaways from the MPS World Summit 2023

Five key takeaways from the MPS World Summit 2023

Neurite extension from motor neurons to myotubes and synapse formation detected by Bungarotoxin staining

Axol Bioscience report on the MPS World Summit 2023

On the 26th June, the MPS World Summit 2023 kicked off in Berlin, bringing together researchers, scientists, and professionals from around the world. Axol CEO, Liam Taylor, and Product Manager, Jan Turner, were in attendance to fly the flag for human iPSCs and Axol’s work developing high-quality, human-relevant cells to fuel microfluidic platforms. In this article, we outline their key highlights and takeaways from the Summit.

Microphysiological systems (MPS) are advanced in vitro platforms that place cells in complex microenvironments that mimic physiological conditions. MPS has actually become an umbrella term comprising a range of platforms; microfluidic devices, 3D co-cultures, organoids, micro-tissues and organ-on-a-chip platforms are all examples of MPS.

The MPS World Summit brought together 1300 industry professionals to discuss the opportunities, advancements, and areas for improvement for the MPS industry.

#1: Microphysiological Systems could be transformative for drug discovery

The drug discovery industry is excited about MPS. With the potential to construct complexhuman-relevant models that closely mimic human physiology in vitro, there is clear value for using these platforms to develop better, safer drugs. In fact there are already a number of consortia including stakeholders from academia, pharma, MPS developers and regulators such as IQ MPS and IMPSS collaborating to enable their use in drug discovery.

Human iPSCs can be used as the “fuel” to generate human-relevant MPS that incorporate patient-derived cells, as well as healthy control donors. You can also combine multiple cell types into complex architectures, enabling cell-cell crosstalk and microfluidics to better recapitulate human physiology.

Ultimately, the benefit lies in closing the translational gap produced by animal models and simple cell cultures. With an R&D spend in the region of $6 billion per drug and failure rates as high as 99.6% for Alzheimer’s therapies, the need for better, more human-relevant models is clear.

The power of MPS technology to emulate genuine human biology under experimental conditions is likely to make many animal experiments redundant.– excerpt from MPS World Summit description

#2: Powerful models need to be fueled by high-quality cells

With a range of potential formats in 2D and 3D, there was great emphasis on the structures and scaffolds themselves, with standardization frameworks such as ISO 22916 entering many discussions. But in order to build truly powerful, human-relevant models, the industry must also focus on the quality, consistency, and standardization of cells used to power them.

With key drivers like the FDA Modernization Act 2.0 and the ISSCR Standards Document, there is a clear effort towards developing best practices in the stem cell space. This is vital to ensuring cells- such as human iPSCs- are developed with an emphasis on functional relevance, batch-to-batch consistency, and quality of end products. Large consortia such as CEN CENELEC and The European Organ-on-Chip Society (EUROoCs) have a pivotal role to play in driving this focus on cell standardization, which will subsequently improve the quality and outputs of MPS themselves.

#3: The industry needs to prioritize pre-clinical diversity

A key panel discussion focused on the importance of pre-clinical diversity, which has been historically under-emphasized. When including only a limited demographic representation in cell samples, there is a risk of failing to include important gene polymorphisms, sex differences, and racial differences in drug discovery.

The general takeaway from this discussion was the importance of starting at the donor cell stage, drawing on a diverse range of patients and healthy control donors. This is something we’ve identified at Axol; we have built our axoLines iPSC library with a 50:50 split of male:female donors, and are looking to develop a ‘Clinical Trial In A Dish‘ for Alzheimer’s Disease using a large pool of donor samples collected by StrataStem.

#4: We must adopt a data-driven approach to MPS platforms

Ultimately, for MPS to effectively accelerate drug discovery, they need to be built with clearly-defined, measurable endpoints (such as electrophysiology). This will ensure the generation of useful quantitative data at the pre-clinical stage, helping to narrow down compound libraries or provide information to support further clinical trials.

An interesting theme during the Summit focused on the amenability of MPS to machine learning/artificial intelligence, which could analyze enormous quantities of data generated from high-throughput models to deliver novel insights for drug discovery. This would present a significant advantage over traditional animal models but would require the development of high-throughput MPS that are amenable to computational analysis.

#5: There is a clear industry demand for high-quality, standardized platforms

Across the entire event, there was one clear message: the drug discovery industry has a clear need for MPS and are excited at the potential implications for reduced cost, risk and failure rates. It was reported at the Summit that 30% of Big Pharma companies have complex in vitro model groups, such as Roche’s Institute of Human Biology, and this is likely to increase as the industry develops greater MPS utility.

With human iPSCs specifically, there is a clear understanding of their value and utility, but with a need to prioritize quality and consistency. This is something we are keen to drive at Axol, given our long-standing history of driving industry standards for iPSC quality and consistency from our manufacturing facility in Roslin.

We’ve applied over a decade of experience to develop high-quality human iPSC-derived cells for use in MPS systems, with expertise across the neurodegenerative and cardiovascular space. Plus, with our custom axoServicesTM offering, we offer in-house R&D project capabilities for platform providers to accelerate your product development and delivery. We’re also looking to collaborate with companies who share our vision, to develop advanced axoModelsTM using human iPSCs.

For more information, click here: Cells for organ-on-a-chip and MPS platforms (axolbio.com)

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