Ground-breaking therapy could herald a cure for Huntingdon’s

As fans may have seen on TV series House, Huntington’s disease is an inherited condition that damages certain nerve cells in the brain. This brain damage gets progressively worse over time and can affect movement, cognition (perception, awareness, thinking, judgement) and behaviour.

Up until now, this heartbreaking disease has had no cure, is usually fatal and worse, is hereditary.  Life expectancy in HD is generally around 20 years following the onset of visible symptoms. Most life-threatening complications result from muscle coordination and, to a lesser extent, behavioral changes induced by declining cognitive function.

However, a revolutionary new treatment could bring hope of a cure to the one in every 10,000 affected by this debilitating disease thanks to what scientists describe as ‘the biggest breakthrough for neurodegenerative diseases for half a century’.

The first drug trials to target the cause of Huntington’s disease was recently undertaken by UCL Institute of Neurology’s Professor Sarah Tabrizi, sponsored by Ionis Pharmaceuticals.

The drug proved successful in lowering the level of the harmful huntingtin protein known to damage the nervous system in 46 patients with early Huntington’s disease at nine study centres in the UK, Germany and Canada.  Patient safety was monitored throughout the study by an independent safety committee and the drug was well-tolerated by the trial participants.  The University plans to extend the trial to more patients – with the lead researcher describing the results as ‘ground-breaking’.

Professor Tabrizi, Director of the UCL Huntington’s Disease Centre, told the BBC:

“I’ve been seeing patients in clinic for nearly 20 years, I’ve seen many of my patients over that time die.

“For the first time we have the potential, we have the hope, of a therapy that one day may slow or prevent Huntington’s disease.  This is of groundbreaking importance for patients and families.”


The research is supported by The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre. The centre is a partnership between UCL and University College London Hospitals NHS Foundation Trust funded by the NIHR to translate scientific breakthroughs into better patient treatments.