The first drug targeting the cause of Huntington’s disease was safe and well-tolerated in its first human trial.
It also successfully lowered the level of the harmful huntingtin protein in the nervous system, results show.
Over a decade in pre-clinical development, this first human trial of huntingtin-lowering drug began in late 2015, led by Professor Sarah Tabrizi from University College London.
The trial involved 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.
Sarah Tabrizi said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families.
“For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated.
“The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”
The results of the trial and plans for the ongoing IONIS-HTTRx programme will be presented in detail at forthcoming scientific meetings and are being prepared for peer-reviewed publication.
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