Huntington's Disease: Experimental Drug Marks Huge Breakthrough In First Human Trial


A groundbreaking trial for Huntington's disease has shown that an experimental drug could slow down the progression of the disease.

Huntington's disease is ruthless and it currently doesn't have a cure. It affects the brain and the damage is irreversible. Current treatments can only help with symptoms, but not slow down its progression.

Experimental Drug Could Slow Down Huntington's Disease Progression

The positive results of this landmark trial spark hope that one day Huntington's disease might have a cure. Researchers at the University College London (UCL) developed an experimental drug which, when injected into the spinal fluid, managed to lower the levels of toxic proteins called huntingtin affecting the brain.

With these breakthrough results, the research team thinks that the deadly disease might one day be stopped. No drug has ever managed to suppress the effects of the Huntington's mutation, which is why this trial is extremely significant and holds great promise.

This was the first trial for Huntington's conducted on human patients and the drug proved to be safe. Trial patients have tolerated it well and it managed to lower the level of huntingtin in the brain.

Huntington's Human Trial

The experimental drug was in pre-clinical development for more than 10 years and this trial on human patients started toward the end of 2015. Professor Sarah Tabrizi of the UCL Institute of Neurology led the trial, and Ionis Pharmaceuticals sponsored it.

The research team conducted the trial at nine facilities in Canada, Germany, and the UK, on 46 people with an early-stage Huntington's disease. Each of the patients enrolled in the trial received four doses of either placebo or IONIS-HTTRx, injected into the spinal fluid so it gets to the brain. The IONIS-HTTRx dose was gradually increased throughout the trial and an independent safety committee closely monitored patient safety from the beginning to the end of the trial.

The trial has now ended and concluded that patients have successfully tolerated IONIS-HTTRx and it had positive effects, so further testing is warranted.

Larger Huntington's Test Trial To Follow

"The results of this trial are of ground-breaking importance for Huntington's disease patients and families," says Tabrizi. "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."

Huntington's is one of the most ruthless, relentless, and devastating diseases, and it's transmitted genetically. It's caused by a mutation in a section of DNA, dubbed the "huntingtin gene."

Children born to a parent with Huntington's have a 50-50 chance of developing the disease. Huntington's typically affects people in their 30s to 40s, and patients usually die within 10 or 20 years after the first symptoms manifest.

Should this experimental drug prove to slow down the disease progression, it could mark the biggest breakthrough in neurodegenerative diseases for the past five decades.

Following this promising study, Roche, a partner of Ionis Pharmaceuticals, agreed to license IONIS-HTTRx and will further develop the drug, as well as handle regulatory requirements, and commercialization. All people who participated in this Huntington's trial will be included in an open-label extension so they can receive more IONIS-HTTRx.

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