Huntington’s Disease Cure Peeks Over the Horizon as New Drug Trial Shows Promising Results

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Considered one of the most debilitating diseases even amongst neuro-degenerative conditions, Huntington’s disease may have finally met its match in the form of Professor Sarah J. Tabrizi of the University College of London’s (UCL) Institute of Neurology.

Director and Lead Researcher at the Huntington’s Disease Centre at UCL, Prof. Tabrizi led the first human trials of a new drug, with highly encouraging results.

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.

– Prof. Sarah Tabrizi.

Currently known under the name Ionis-HTTRx, the groundbreaking drug was shown not only to be safe for humans, but most significantly, it lowered the levels of the mutant huntingtin protein, the cause of the damage Huntington’s disease presents with.

In fact, the drug’s effectiveness is such that pharmaceutical giant Roche has licensed the drug and will initiate a second, much larger trial, details of which remain to be announced in 2018.

Ionis-HTTRx was developed by Roche partner Ionis Pharmaceuticals over the course of 10+ years, and started its first human trials more than two years ago, in late 2015.

The trial involved 46 patient participants in nine medical study centers in Canada, U.K. and Germany, who received four increasing doses of the new drug (or a placebo). Delivered into the spinal fluid to most efficiently reach the brain, the first trial has shown it to lower huntingtin levels in the nervous system – a historic first.

Professor Sarah J. Tabrizi, Director and Lead Researcher at the Huntington’s Disease Centre at the Institute of Neurology, University College of London
Professor Sarah J. Tabrizi, Director and Lead Researcher at the Huntington’s Disease Centre at the Institute of Neurology, University College of London | Image credit: HDC

2017 saw a number of breakthroughs in the understanding of Huntington’s disease. Back in mid-June, a joint research team from UCL and Cardiff University announced the identification of a genetic modifier that plays a role in disease progression. A better understanding of Huntington’s progression is a crucial step in developing more effective therapies and treatments.

Before that, early June saw the announcement of the discovery of a blood test that can predict the onset of Huntington’s. The test can also track disease progression, and was authored by Lauren Byrne and Dr. Ed Wild, among other scientists. Both studies were conducted by researchers at the UCL’s Huntington Disease Center, and won separate “Insight of the Year” awards from the Huntington Study Group.

The implications of Ionis-HTTRx’s effectiveness reach beyond better treatments and a cure for Huntington’s – the breakthrough could revolutionize the entire field of neurodegenerative diseases and their therapies and treatments, including dementia.


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