A new Drug responsible for lowering Huntington’s disease protein
The first drug targeting the reason for Huntington’s disease was safe and well-tolerated in its first real human trial led by UCL researchers. It successfully reduced the amount of the damaging Huntington’s necessary protein in the Nervous system.
After over ten years in pre-clinical development, this first human being trial of Huntington-lowering medication began in late 2015, led by Professor Sarah Tabrizi (UCL Institute of Neurology) and sponsored by Ionis Pharmaceuticals.
Professor Tabrizi, Director of the UCL Huntington’s Disease Center and IONIS-HTTRx Global Chief Investigator, 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 trial included enrolling 46 patients with early on Huntington’s disease at nine study centers in the UK, Germany and Canada. Each patient received four doses of either IONIS-HTTRx or placebo, injected into the spinal liquid to enable it to reach the brain. As the stage 1/2a trial progressed, the dose of IONIS-HTTRx was increased several times in line with the ascending-dose trial design.
The trial confirms that IONIS-HTTRx was well-tolerated by the trial members and its safeness profile supports further trials in patients. A significant unknown was whether the trial would show that IONIS-HTTRx could lower the amount of mutant Huntington necessary protein in the nervous system. Using an ultra-sensitive assay, concentrations of the necessary protein were assessed in each patient’s spinal fluid before and after treatment.
As hoped, IONIS-HTTRx– produced significant, dose-dependent decreasing of the amount of mutant Huntington – the very first time the protein known to cause Huntington’s has been reduced in the nervous system of patients.
Due to these successful effects, Ionis’ partner, Roche, has exercised its option to certificate IONIS-HTTRx and assumes responsibility for further development, regulatory activities and commercialization activities. Meanwhile, Ionis announced in June that patients in the completed trial would be offered a place within an open-label extension to receive IONIS-HTTRx.
The research is reinforced by The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre. Partnership between UCL and University College London Hospitals NHS Foundation Trust funded by the NIHR to translate scientific breakthroughs into better patient treatments.
Source- UCL News