Tuesday, 13 May 2014

Model Neurological Disease in a Dish

Over one billion people worldwide are affected by brain disorders such as Alzheimer’s disease, autism, stroke and traumatic brain injury (1). Up to now, the research on these diseases has relied heavily upon animal models. Genetically modifying animals to mimic human brain disease is an expensive and time-consuming process. More notably, the animal models often do not truly recapitulate the relevant human pathology and drug response.
Thus, frequently, drugs that have appeared to be “promising” in animal tests were not effective in patient trials. Lack of appropriate preclinical models for studies of brain diseases and drug efficacy predictions is now recognised as a major cause of new drug clinical trial failure. This has made the development of medications for treatment of brain disorders extremely arduous and highly expensive.

Human cell-based disease models are becoming key tools for biomedical research, especially for disease and drug mechanism studies. Automation of cell culture, imaging and assays has made high throughput data generation and analysis possible with many drug-targeting cell types, such as cancer cells. However, human neural cell types are very difficult to obtain, isolate and expand in the lab. These obstacles have significantly hindered the development of neural cell-based models for neurological diseases. 

Human pluripotent stem cells can proliferate indefinitely and have the potential to generate all the cell types of the human body, including neural cell types. In the past decade, these stem cells have been isolated from live human embryos. The recent Nobel Prize-winning Cellular Reprogramming technology (2) has revolutionised the way we obtain these stem cells. It provides the opportunity to derive these stem cells in an ethical way, from easily accessible adult cell types, such as blood cells or skin fibroblasts. 

Axol has developed a proprietary stem cell technology to instruct these pluripotent stem cells to produce high quality brain neural cell types. This and the reprogramming technology together provide us with the capacity to produce a variety of brain neural cell types at industrial quantity from any individual, including patients afflicted with brain diseases. With this capacity, we can now offer the research community an affordable and easily accessible platform for developing human cellular models of neurological diseases. These cellular models can be used to complement existing animal models in drug discovery, allowing researchers to identify new drugs that show efficacy on the disease-relevant human neural cells rather than simply on animal models. They can also be used in toxicity tests in order to prevent unsafe drugs from entering into clinical trials, thereby reducing the risk to the study group population and improving the chances of a successful outcome.

The research councils’ increasing emphasis on translational medicine-related projects has triggered the ever-rising demand for these types of cell products. More and more labs are now looking for the link between their research and human biology, diseases and therapies. For instance, scientists are eager to find cost-effective ways to study the unique features of human neural tissue formation and function, and confirm their research findings from model organisms in human context. The development of effective cell therapies for neural degenerative disorders and nervous system injuries is another research area of intense focus. The current paucity of transplantable human neural cells has been driving the demand for preclinical studies of human stem cell-derived neural cells for transplantation purpose.

Axol products are distributed in the UK by Caltag Medsystems.


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For further information on any of these products, please contact Caltag Medsystems by email or call 01280 827460.

References:
1. Neurological Disorders: Public Health Challenges, World Health Organization Report 2007, ISBN 9241563362
2. 2012 Nobel Prize in Physiology or Medicine; Mature cells can be reprogrammed to become pluripotent: Dr. John B. Gurdon and Dr. Shinya Yamanaka

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