Axol and StrataStem Announcement
Axol and StrataStem announce collaboration on large-scale Alzheimer’s iPSC cohort
The agreement signifies the first steps in building powerful in vitro Alzheimer’s models for drug discovery and patient stratification
In their mission to make more powerful human disease models, Axol Bioscience and StrataStem have announced a collaboration agreement 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.
- Axol Bioscience, the experts in human induced pluripotent stem cell (iPSC) technology, have signed a collaboration agreement with StrataStem to access and commercialize a large-scale collection of Alzheimer’s Disease (AD) patient samples
- Using Axol’s expertise and technology, the company will reprogram these patient samples into iPSCs, which can then be differentiated into a wide range of brain cells such as neurons and neuroinflammatory cells
- These brain cells (which are implicated in AD pathophysiology) can then be grown in vitro, in a manner that models the human brain
- This novel large cohort approach using stem cell technology will enable the creation of a ‘clinical trial in a dish’ platform, accessible for drug-discovery companies to engage in drug development and patient-stratification studies
The current landscape
Dementia is a growing public health concern which affects over 50 million people globally, a total which is projected to grow to more than 150 million by 2050. Alzheimer’s disease (AD) is the most common type of dementia, predominantly affecting those 65 years and older, and is characterized by a global decline in cognition and an inability to perform daily activities.
We have previously demonstrated the ability to create ‘co-cultures’ of three or four brain cell types, differentiated from iPSCs, to generate sophisticated in vitro models that exhibit key hallmarks of Alzheimer’s Disease.
While these models have proven useful and effective, they do not adequately address the inherent complexity of AD. The phenotypic and genotypic variability in AD means early diagnosis and patient stratification is challenging. With the lack of effective diagnostic methods (current estimates of 25% misclassification) and no single genotypic marker, there is an urgent need for larger-scale models with additional power.
How the large cohort 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.
Donated samples include information on:
- Disease state
- Medical and lifestyle history
- Family health summaries
The power of a large-scale ‘clinical trial in a dish’
Representing a physiologically relevant, yet diverse patient cohort in an in vitro format is the key to producing a powerful platform.
Selection of the ‘right’ population for clinical trials is vital for drug development success; due to the challenges of Alzheimer’s diagnosis and the inherent disease variation, this has proven challenging in the neurodegenerative disease space.
The ability to better stratify and then select individuals for inclusion in clinical trial may unlock drug progress, reduce failure rates and off-target effects, and bring new insights into AD cohorts.