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Breakthrough in Huntington's disease research


Biomedical scientists at University College London (UCL) have successfully trialled an experimental drug that that safely lowers the level of harmful protein in the nervous system.


The ground-breaking research, which has been described by biomedical scientists as the biggest development in neurodegenerative diseases for half a century, brings new hope for the thousands of people who have Huntington's disease (HD), which affects about 12 in every 100,000 of UK residents.

In the trial, which began in 2015, 46 patients were each given four doses of IONIS-HTTRx or a placebo. It was unknown whether the drug could lower the level of mutant huntingtin protein in the nervous system. However, not only did it lower the level of toxic proteins in the brain, it was also found to be well tolerated by all recipients. As a result, pharmaceuticals giant Roche has now exercised its option to license the drug. In addition, the research partner Ionis has announced that all patients in the completed trial will be offered a place in an open-label extension to receive IONIS-HTTRx.

The trial was led by Professor Sarah Tabrizi, a biomedical science specialist and Director of the UCL Huntington's Disease Centre, which opened on 1 March 2017. Professor Tabrizi and her co-director, Professor Gill Bates, are world leaders in bench-to-bedside HD research, Even before this latest breakthrough, they had already made considerable contributions to the search for a cure for the disease in a wide range of areas, including gene identification, animal modelling, therapeutic target validation and experimental medicine. This latest research is supported by the National Institute for Health Research (NIHR) UCL Hospitals Biomedical Research Centre, a partnership between UCL and UCL Hospitals NHS Foundation Trust.

Caused by an inherited faulty gene, HD has a devastating effect on patients and their families. Brain damage gets progressively worse and can affect movement, cognition and behaviour. Currently, there's no cure for the disease and its progress can't be reversed or slowed down. However, some of the symptoms, such as involuntary movement and mood swings, can be controlled by drugs. In addition, speech and language therapists and occupational therapists can help patients with communication and day-to-day living.

"The results of this trial are of ground-breaking importance for Huntington's disease patients and families, " said Professor Tabrizi. "For the first time a drug has lowered the level of toxic disease-causing protein in the nervous system, and the drug was safe and well tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression."
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