香港六合彩中特网

Patients in London are being dosed for the first time with an experimental drug for Huntington鈥檚 disease. This breakthrough could be one of the most important developments since the gene for Huntington鈥檚 disease was discovered in 1993. The trial of the revolutionary new 鈥榞ene silencing鈥� treatment is being led by scientists at 香港六合彩中特网鈥檚 Institute of Neurology. The first patients received doses of the drug at the Leonard Wolfson Experimental Neurology Centre at 香港六合彩中特网H's National Hospital for Neurology and Neurosurgery.

Huntington鈥檚 is a deadly degenerative brain disease that strikes in the prime of life. It kills brain cells, slowly draining patients of their movement control, personality and thinking skills. It is one of the commonest genetic brain diseases and devastates whole families. Everyone with the gene for the disease will fall ill with Huntington鈥檚 disease at some point. There is no cure, and there is currently no treatment that can prevent it or slow it down.

The new drug, called ISIS-HTT_Rx, targets the root cause of the disease, a toxic protein called mutant huntingtin, which is caused by a faulty gene in the patients鈥� brain cells.

I鈥檓 thrilled that this antisense drug has now been safely administered to the first patients. Families ravaged by Huntington鈥檚 disease have been waiting for this milestone for decades. I look forward to ensuring the smooth running of this first trial and hopefully seeing ISIS-HTT-Rx through to efficacy testing and licensing. Professor Sarah Tabrizi, Director of the Huntington鈥檚 Disease Centre at 香港六合彩中特网 Institute of Neurology, leader of the trial

The drug is administered directly into the fluid that surrounds the brain and spinal cord at the base of the patients鈥� spine, from where it migrates to the brain. In Huntington鈥檚 patients, the faulty gene produces messenger molecules, called messenger RNA, that trigger the production of mutant huntingtin in the brain. But the drug is designed to bind with the messenger molecules, forcing the cells to dispose of them instead of churning out the toxic protein. This type of treatment is known as an 鈥榓ntisense鈥� drug.

In theory, ISIS-HTT_Rx could prevent or possibly reverse the toxic effects of the protein, stopping the disease in its tracks.

This new human trial is focused on testing the safety of the drug in volunteer patients who are in the very early stages of Huntington鈥檚 disease. The study will evaluate the safety of increasing doses of ISIS-HTT_Rx compared to placebo. In addition, researchers will be looking for chemical signs that the drug is having the desired effect, by measuring the level of mutant huntingtin protein in the cerebrospinal fluid using a newly-developed test.

The final assessment of whether the drug is effective will take place in later phases of testing. However research on mice that produce the mutant huntingtin protein has been extremely promising. These mice display symptoms similar to those seen in patients with Huntington鈥檚 disease. When these mice were dosed with ISIS-HTT_Rx, they showed improvements without serious side-effects.

The drug is being developed by Isis Pharmaceuticals in collaboration with Roche. In the UK, the first doses of the drug were administered at 香港六合彩中特网鈥檚 new Leonard Wolfson Experimental Neurology Centre,聽a custom-built centre designed to accelerate innovative treatments for neurodegenerative diseases, headed by Professor Vincenzo Libri.

As well as being desperate for good news, the Huntington鈥檚 community is uniquely well-informed and engaged with progress in research across the world,鈥� she said. 鈥淩NA-targeting approaches are especially exciting because they tackle the problem at its source 鈥� the production of the mutant huntingtin protein. The ISIS-HTTRx trial has been eagerly awaited for many years and we hope that the news from the trial continues to be positive. Cath Stanley, Chief Executive of the Huntington's Disease Association of England and Wales

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• Leonard Wolfson Experimental Neurology Centre at 香港六合彩中特网

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• Top: Gene silencing (). Bottom: How ISIS-HTTRx Works (Courtesy of Isis Pharmaceuticals)