Landmark Clinical Trial Begins for Promising Anti-Sense Gene-Silencing Drug to Block Production of Mutant Huntingtin Protein That Causes Huntington’s Disease; Drug Developed by Isis Pharmaceuticals & Roche

On October 19, 2015, it was announced that patients in London are being dosed for the first time with an innovative new experimental drug for Huntington’s disease. This possible clinical breakthrough could be one of the most important developments since the gene for Huntington’s disease was discovered in 1993. The clinical trial of the revolutionary new “gene-silencing” treatment is being led by scientists at the University College London’s (UCL’s) Institute of Neurology. Huntington’s 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 most common genetic brain diseases and it devastates entire families. Everyone with the mutated gene for the disease will fall ill with Huntington’s disease at some point. The disease is dominantly inherited and there is currently no cure and there is also no treatment that can prevent it or slow it down. The new drug being tested is called ISIS-HTTRx and it targets the root cause of the disease, a toxic protein called mutant huntingtin (HTT), which is coded for by a faulty gene (mutant HTT gene) in the patients’ brain cells. The drug is administered directly into the fluid that surrounds the brain and spinal cord at the base of the patients’ spine, and from there the drug migrates to the brain. In Huntington’s patients, the faulty HTT gene codes for messenger molecules, called messenger RNA, that trigger the production of mutant huntingtin protein the brain. But the drug is designed to bind with the messenger RNA molecules coded for by the faulty HTT gene, forcing the cells to dispose of them instead of the mRNA churning out the toxic protein.
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