Drug lowers deadly Huntington’s disease protein

The first drug targeting the cause of Huntington's disease was safe and well-tolerated in its first human trial led by UCL scientists. It successfully lowered the level of the harmful huntingtin protein in the nervous system. After over a decade in pre-clinical development, this first human trial of huntingtin-lowering drug began in late 2015, led by Professor Sarah Tabrizi (UCL Institute of Neurology) and sponsored by Ionis Pharmaceuticals. The trial involved enrolling 46 patients with early Huntington's disease at nine study centres in the UK, Germany and Canada. Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain. As the phase 1/2a trial progressed, the dose of IONIS-HTTRx was increased several times according to the ascending-dose trial design. Patient safety was monitored throughout the study by an independent safety committee.