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Since the discovery of the gene for Huntington’s disease in 1993, the development of experimental new drug ISIS-HTTRx, has been one of the most important breakthroughs. For the first time, in London, the patients of Huntington’s disease are being dosed with this experimental drug. The first human trial of this revolutionary new gene-silencing treatment is being carried out at UCL’s new Leonard Wolfson Experimental Neurology Centre, a custom-built centre designed to accelerate innovative treatments for neurodegenerative diseases, which is located at UCLH's National Hospital for Neurology and Neurosurgery, UK.

Huntington’s disease, a deadly degenerative genetic brain disease, kills brain cells and affects patient’s movement control, personality and thinking skills. Currently, this deadly disease that strikes the prime of life, has no cure or has no treatment to prevent it or to slow it down. A faulty gene in the patients’ brain cells, produces messenger molecules, called messenger RNA, that trigger the production of toxic protein called mutant Huntington, which is the root cause of the Huntington’s disease. The new drug, called ISIS-HTTRx, is designed to target this root cause of the disease. The drug, when administered directly into the fluid that surrounds the brain and spinal cord at the base of the patients’ spine, it migrates to the brain and binds with the messenger molecules, forcing the cells to dispose them instead of churning out the toxic protein. These type of drugs are called ‘antisense drugs’.

This new human trial evaluates the safety of increasing doses of ISIS-HTTRx compared to placebo in volunteer patients who are in the very early stages of Huntington’s disease. Researchers measure the level of mutant Huntington protein in the cerebrospinal fluid and make their assessment. However, research on mice that displayed symptoms similar to those seen in patients with Huntington’s disease, showed extremely promising results. Families ravaged by Huntington’s disease have been waiting for this milestone for decades. The ISIS-HTTRx trial has been eagerly awaited for many years and researchers hope that the news from the trial continues to be positive.