Biotechnology

A ‘deep-dish clinical trial’ for Alzheimer’s disease

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

Leveraging its stem cell knowledge, Axol will reprogram these patient samples into induced pluripotent stem cells (iPSCs) which can then differentiate into various brain cells, including neurons and neuroinflammatory cells. Grown in vitro, these cells could provide patient-specific human brain models from large cohorts of AD patients – to create ‘clinical trials in a dish.’

Seeking to accelerate the search for effective treatment for AD, this work specifically focuses on sporadic AD (sAD), the most common form of the disease, which accounts for more than 95% of all cases. sAD is not specifically related and is caused by a combination of genetics, environment, and a person’s lifestyle.

StrataStem donor material collections, including fibroblasts and blood samples, have been obtained from fully consenting sAD patients and healthy donors. All materials are supported by extensive longitudinal data on disease state, medical history and lifestyle, as well as family health summaries.

By manufacturing iPSCs and generating differentiated brain cells from this large-scale Alzheimer’s cohort, Axol aims to enable pre-clinical stratification of patients for AD treatments, enabling drug discovery companies to select the most responsive patients to trial.

About the Axol Bioscience approach

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

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

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

Chris Ward, CSO and co-founder, StrataStem, added: “We are excited to enter into this collaboration with the team at Axol. The recent US Federal Food and Drug Administration (FDA) Modernization Act 2.0 has paved the way for the use of cell-based tests to investigate drug safety and efficacy, including this innovative ‘in-dish clinical trial’ approach. We expect these advances to reduce drug development risks and speed up the process, while providing valuable cost and time savings.

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

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