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Case study: Advances in cell therapy for Huntington's Disease

Find out more about the NIHR Dementia Translational Research Collaboration

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"This study has enabled the NIHR to develop new ways of thinking about trials of novel cell therapies for diseases of the brain."

A safety study involving the transplantation of human fetal striatal tissue into the striatum of four patients with Huntington’s disease has demonstrated that the procedure is safe and feasible.

The current lack of treatment for Huntington’s disease has led to efforts by the NIHR Cambridge Biomedical Research Centre to translate cell therapy advances into disease modifying treatments. A safety study involving the transplantation of human fetal striatal tissue into the striatum of five patients with Huntington’s disease has demonstrated that the procedure is safe and feasible.

Stereotaxic tissue placement following pre-operative magnetic resonance imaging was performed in Cambridge, UK by investigators from the NIHR Cambridge Biomedical Research Centre (NIHR Cambridge Dementia BRC) as part of a broader UK network. Cambridge-based protocols for the screening of human fetal tissue for potential pathogens have also been developed, with clinical trials planned in Parkinson’s disease. Cambridge surgeons have now grafted five patients and the long term follow up of which has been published in the Journal of Neurology, Neurosurgery & Psychiatry.

Vital research

Huntington’s disease affects an estimated 6,500 to 8,000 people in the UK. This is an incurable inherited disease that causes the progressive loss of nerve cells in the brain. One of the major sites where this occurs is in a structure called the striatum which sits deep within the brain. Research during the 1980’s and 1990’s showed that it was possible to transplant fetal striatal tissue into the brain of animal models
of Huntington’s disease, where it could survive and make connections with the host brain. The ability of this fetal tissue to survive and replace that lost in the experimental model was paralleled by improvements in some of the behaviours in these animals. In 2000, given this and the fact that there are no drugs or therapies that can stop or arrest the disease process in these patients, the NIHR Cambridge Dementia BRC team undertook a similar study in human patients with Huntington’s disease.

Study findings

The study involved patients with well established Huntington’s disease, where human fetal striatal tissue was transplanted into the striatum on one side, and then the other. The procedure was found to be safe, with no major complications from the surgical implantation of the tissue. The NIHR Cambridge Dementia BRC team found no major placebo effect from the operation and also saw no major improvements in the patients following the transplant. This suggests that this treatment does not work in Huntington’s disease, at least using this protocol.

The project finished and clinical data from the project was published in 2013, and the post mortem data on one of the grafted patients was recently published in the Annals of Neurology.

Patient Impact

The study is important as it has shown that it is possible to transplant cells into the brains of patients with Huntington’s disease and that there is no major placebo effect from doing so. The treatment has shown no long term side-effect but then again no benefits, which may relate to the protocol used and / or the fact that Huntington’s disease affects many other parts
of the brain, not just the striatum.

Professor Roger Barker, Theme Lead for Dementia and Neurodegenerative Disorders at NIHR Cambridge Biomedical Research Centre commented: “This study has enabled the NIHR to develop new ways of thinking about trials of novel cell therapies for diseases of the brain. It will help decide how the NIHR sets about formulating future stem cell-based trials for chronic neurodegenerative diseases of the brain. While this study concluded that using this specific protocol resulted in no major improvements, we have advanced our understanding of potential treatment methods for Huntington’s.”

Nest steps

The project finished and clinical data from the project was published in 2013, and the post mortem data on one of the grafted patients was recently published in the Annals of Neurology. This therapy is now being taken forward through a stem cell approach and a new initiative has been set up to those globally; SC4HD, which had its first meeting in LA in May 2018.

 


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