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Neural stem cell grafts protect neurological function in Batten disease mice

13 October 2009

Dr Jonathan Cooper from the Department of Neuroscience, Institute of Psychiatry at King’s College London and colleagues in the US have published preclinical data demonstrating for the first time that transplantation of human neural stem cells delays the loss of motor function and protects brain cells from dying in a mouse model of the infantile neuronal ceroid lipofuscinosis (NCL). NCL is more commonly referred to as Batten disease and is a fatal progressive neurodegenerative disorder in children and young adults.

This work was performed in collaboration with colleagues from StemCells Inc, the Stanford University School of Medicine and The Salk Institute for Biological Studies and was recently published in a paper: entitled: Neuroprotection of Host Cells by Human Central Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis in the journal Cell Stem Cell. Dr Cooper additionally acted as a consultant to StemCells Inc.

Dr Cooper said: 'These are exciting results that provide proof of principle that human neural stem cells can impact the progression of disease in a mouse model of NCL, paving the way to testing if these cells can also improve the quality of life in patients suffering from this devastating disease.'

The study highlights a novel neuroprotective approach to treating neurodegenerative diseases and the therapeutic potential of neural stems cells. In this research, these cells were transplanted in a mouse model of infantile NCL and compared to a control (non-transplanted) group of mice with the disease. The transplanted cells engraft, migrate throughout the brain and continuously secrete the lysosomal enzyme that is missing in this form of NCL, which is needed to process cellular waste and keep neurons functioning and healthy. Compared with untreated, the NCL mice that received the transplanted neural stem cells showed significantly less build up of cellular waste, less death of neurons and delayed loss of motor function.

This preclinical study lead to Stem Cell Inc sponsoring a phase I safety trial of its proprietary human neural stem cells (HuCNS-SC™) in children with infantile and late infantile Batten disease at Oregon Health & Science University's Doernbecher Children's Hospital, which was completed earlier this year. This was the first trial of human neural stem cells in any human neurodegenerative disorder.

The full paper can be accessed here: www.cell.com/cell-stem-cell/abstract/S1934-5909(09)00231-8

Dr Cooper’s web page re his work with Batten Disease can be found here: http://www.iop.kcl.ac.uk/departments/?locator=382

 

 

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