A cure for Huntington's disease could be in the pipeline following the results of a groundbreaking new drug trial.For the first time, scientists have been able to correct the defect that is responsible for the devastating neurodegenerative disease.The results, hailed as the 'biggest breakthrough in 50 years', saw 46 patients of the incurable disease given an experimental drug.University College London researchers discovered the pill lowered their levels of toxic proteins in the brain.The drug, dubbed a 'tremendous step forward', was also found to be safe - despite fears it could lead to meningitis.Professor Sarah Tabrizi, lead researcher told the BBC: 'I've been seeing patients in clinic for nearly 20 years, I've seen many of my patients over that time die.'For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington's disease.'Professor Tabrizi, director of UCL's Huntington's Disease Centre, added: 'This is of groundbreaking importance for patients and families.'Huntington's disease is a genetic disorder that affects the central nervous system and leads to involuntary movements, difficulty talking and memory loss.The condition, which affects 8,500 adults in the UK and around 30,000 in the US, occurs as a result of a fault in the huntingtin gene.On average, patients live for between 10 and 20 years after their diagnosis.The huntingtin protein itself is vital for the development of the brain, but the genetic error leads to it instead killing brain cells.Huntington’s disease is an illness caused by a faulty gene in your DNA (the biological ‘instructions’ you inherit which tell your cells what to do). If you have Huntington’s, it affects your body’s nervous system – the network of nerve tissues in the brain and spinal cord that co-ordinate your body’s activities. Huntington’s can cause changes with movement, learning, thinking and emotions. Once symptoms begin, the disease gradually progresses, so living with it means having to adapt to change, taking one day at a time. Living with Huntington’s disease can be very challenging. Getting the right information and support is vital and we’re here to help. Huntington’s is not something you can catch; it is inherited. Every child conceived naturally to a parent who carries the Huntington’s gene has a 50% chance of inheriting it.The new drug works by silencing the faulty huntingtin gene - stopping the creation of damaged proteins in the brain, the BBC reports.Having proved successful on animals, the British researchers looked to investigate the drug's effects on human patients.For the trial, the patients were injected with the drug into their spinal fluid at the National Hospital for Neurology and Neurosurgery.Alongside its benefits at reducing the amount of huntingtin, the volunteers were found to tolerate the drug well.However, the team remain adamant that more research is needed to prove that lowering levels of huntingtin could lead to a possible
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