Clinical trial in a dish
We expect this advancement to de-risk drug development and expedite the process, while also providing valuable cost and time savings
Chris Ward, CSO and Co-founder of StrataStem
Diverse cohort-scale models 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 allows 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 challenged 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.
Building a large-scale Alzheimer’s model: our collaboration with StrataStem
We recently announced our collaboration with StrataStem involving a large-scale library of human iPSCs, derived from patients with Alzheimer’s Disease. This signifies the first steps in generating better Alzheimer’s Disease models to support drug development and patient stratification.
Watch this video to hear from Duncan Borthwick (Head of Sales and Marketing, Axol) and Chris Ward (CSO and Co-founder, StrataStem) on our collaboration with StrataStem.
Read the full press release here: Axol and StrataStem announce collaboration.
How the “clinical trial in a dish” model works
Using patient samples obtained by StrataStem, Axol will reprogram patient cells into iPSCs. These human iPSCs, which are pluripotent, can then be differentiated into a wide range of brain cells such as neurons and neuroinflammatory cells.
Research to-date has identified several cell types implicated in the pathophysiology of Alzheimer’s Disease, which will form the target end cells produced from the iPSCs. These cells can then be grown in vitro, in a manner that models the human brain environment.
Taking multiple cells from a wide range of donors will enable representation of a large cohort, creating a “clinical trial in a dish” platform. This has enormous potential for drug discovery companies, providing the variety and density of cells for patient stratification and drug development.
The regulatory perspective
When the FDA Modernization Act 2.0 was signed in late 2022, it invited biopharma to embrace alternatives to animal models, such as iPSC-based cell technologies.
There is clear support from regulatory bodies in the pursuit of more human-relevant in vitro disease platforms (such as clinical trial in a dish models). In our interview with regulatory expert, Simon Hoffman, we discussed the implications of the Act for drug discovery, including patient selection and patient stratification.
You can read the full interview here: Simon Hoffman interview.
There are numerous implications… for pharmaceutical companies and change is inevitable; those who embrace the change will thrive.
Simon Hoffman, principal quality assurance and regulatory affairs consultant.
