‘Clinical trial in a dish’ for Alzheimer’s disease

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induced pluripotent stem cells ipsc Alzheimer's

Axol Bioscience Ltd., a pluripotent stem cell technology provider for drug discovery, has signed an agreement with StrataStem to access and commercialize its collection of Alzheimer’s disease (AD) patient samples. 

Harnessing its stem cell knowledge, Axol will reprogram these patient samples into induced pluripotent stem cells (iPSCs) that can then be differentiated into a wide range of brain cells, including neurons and neuroinflammatory cells. Grown in vitro, these cells can provide patient specific human brain models from a large cohort of AD patients – to create a ‘clinical trial in a dish.’

Seeking to accelerate the search for effective treatments of AD, the work specifically focuses on sporadic AD (sAD), the most common form of the disease, which accounts for more than 95% of all cases. sAD has no specific family link and is caused by a combination of genetics, the environment and a person’s lifestyle. 

StrataStem’s collection of donor materials, including fibroblasts and blood samples, has been obtained from fully consenting sAD patients and healthy donors. All materials are supported with extensive longitudinal data on disease state, medical and lifestyle history, and family health summaries. 

Through the creation of iPSCs and resulting differentiated brain cells from this large-scale Alzheimer’s cohort, Axol aims to enable patient stratification prior to clinical trials for AD treatments, allowing drug discovery companies to select the most responsive patients for trial. 

About Axol Bioscience’s approach

Axol’s in vitro approach offers an effective alternative to existing in vivo methods, with the potential to reduce the risk, costs, and timelines of drug discovery and development for AD, with more relevant models of disease.

Liam Taylor, CEO of Axol Bioscience, said: “Here at Axol, we are committed to using iPSCs to make better in vitro models of human disease, expanding our understanding and de-risk drug development. This exclusive agreement with StrataStem is a major strategic move for Axol, bringing us to the forefront of AD drug discovery and development. By commercializing this extensive iPSC library, our drug discovery customers can easily access in vitro models for sAD and interrogate potential mechanisms of the disease more fully.

“Patient stratification at the preclinical stage of drug development is an exciting concept, and we are proud to be among the first to actively source panels of patient samples with comprehensive longitudinal data to do this.”

Chris Ward, CSO and co-founder, StrataStem, added: “We are delighted to be entering this collaboration with the team at Axol. The recent U.S. Federal Food and Drug Administration Agency (FDA) Modernization Act 2.0 has paved the way for the use of cell-based assays to investigate drug safety and efficacy, including this innovative ‘clinical trial in a dish’ approach. We expect this advancement to de-risk drug development and expedite the process, while also providing valuable cost and time savings.

“We are indebted to the patients who continue to support us in advancing our understanding of AD towards the development of more effective treatments. Our collaboration with Axol will massively accelerate this process and enable researchers to better understand how and why the disease develops, help improve the accuracy of diagnosis and will undoubtedly identify new therapeutics for the treatment of the disease.”

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