Close Ad

Huntington’s Disease Cure Peeks Over the Horizon as New Drug Trial Shows Promising Results
researchers-get-close-to-cure-huntingtons-disease
Uplifting News

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

researchers-get-close-to-cure-huntingtons-disease

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\u2019s Disease Centre at the Institute of Neurology, University College of London

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.

Hot Stories

Leonardo DiCaprio Told Kate Winslet To “Let The Fat Girl Thing Go”
Leo Dicaprio Told Kate Winslet To “Let The Fat Girl Thing Go” …

Forced to feel ashamed for her weight and appearance, Kate Winslet struggled with her body image for years. The media loved to tease her for being “the fat girl,” but there was ONE PERSON who saw the real her. What did Leo see in Kate? And what important message does Kate have for young women everywhere?


Keep ReadingShow less
Videos
Older woman with glasses speaking on the phone and younger woman speaking on the phone.

Mother-in-Law Routinely Tricks Woman With This Phone Hack

Even though we’re told we shouldn’t care what other people think of us, deep down, many of us still do. Wanting to be accepted and loved is natural, and it can be devastating to hear someone talk badly about us behind our backs.

However, hearing someone say nice things about us when they don’t think we’re listening can be incredibly uplifting. One mother recognizes that, which is why she routinely "forgets" to hang up on her daughter-in-law.

Keep ReadingShow less
Uplifting News
Woman with three kids and a woman with her three adult children.

40-Year-Old Mother of 3 Goes From Inmate to Princeton Intern

Facebook / Mary McCrary

Life can take us down unexpected and scary roads. Sometimes, we don’t know how to get off those roads and begin the climb to a better future. As this mom proved, however, anything is possible with a second chance and a little determination.

Keep ReadingShow less
Uplifting News