This Huntington’s Breakthrough May Slow Disease Progression

Exciting results from human drug trials suggest that scientists may have discovered a treatment for one of the most debilitating brain conditions: Huntington’s disease. 

The BBC reports on the recent research:

An experimental drug, injected into spinal fluid, safely lowered levels of toxic proteins in the brain.

The research team, at University College London, say there is now hope the deadly disease can be stopped.

Experts say it could be the biggest breakthrough in neurodegenerative diseases for 50 years.

The trial began in 2015 and involved 46 patients with early-stage Huntington’s at nine study centers in the UK, Germany and Canada. Half of the participants were given a drug known as IONIS-HTTRx, which targets a protein thought the be the driver of Huntington’s disease. This drug was administered via injection directly into spinal fluid. Meanwhile, the control group received a placebo. As the trial progressed, the initial four doses were increased several times over.

Researchers found that the drug was effectively able to reduce Huntington’s disease-driving proteins without causing fatal meningitis — an initial concern.

Following this trial, the drug can go on to the next step of safety testing and treatment exploration — an encouraging development, considering that Huntington’s disease has no known cure.

What is Huntington’s disease?

According to the Huntington’s Disease Association, Huntington’s disease is a progressive brain disorder caused by a defective gene. The disease occurs because the defective gene codes for a protein — dubbed “huntington” after George Huntington who first described the disease – that alters brain function.

These changes can manifest in several ways, including a loss of motor function control. People with Huntington’s may experience involuntary movements of their arms, legs and head, which can later progress to a near-total loss of meaningful autonomous movement.

Meanwhile, a marked and progressive loss of reasoning and cognitive skills can impair memory and future planning. Huntington’s also produces a range of mental health problems, including outbursts of anger and aggression, depression, anxiety and obsessive compulsive behavior.

While the disease can impact anyone, men tend to develop Huntington’s more frequently than women. The average age for onset is between 30 and 50 years of age, though it can be much younger. And unlike people with diseases triggered by lifestyle or environment, those who have the Huntington’s gene will definitely develop symptoms at some point in their life.

Why isn’t there a cure?

Put simply, scientists haven’t had the technology or the understanding to correct this genetic fault. CRISPR gene editing may offer a potential solution, but human applications are still a long way off.

Perhaps one of the most frustrating aspects of Huntington’s research has been scientists’ inability to slow its progress. And given that children of Huntington’s sufferers have a 50 percent likelihood of also carrying the gene, this disease’s burden can be terrible.

That’s why the prospect of this new research is exciting. Although the IONIS-HTTRx treatment may not cure Huntington’s, it has the potential to slow or halt symptoms before the disease becomes debilitating.

Dr. Sarah Tabrizi, director of the UCL Huntington’s Disease Centre, explained:

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.

Despite the seriousness of the condition, people with Huntington’s disease can live enjoyable lives, and it can take many years for the worst symptoms to develop.

Even so, it’s welcome news that a drug could potentially slow down or stop the disease in its tracks. And Huntington’s sufferers and their care teams certainly hope that this limited trial will translate into a first-of-its-kind treatment option.

Photo credit: Thinkstock.

50 comments

Marie W
Marie W5 months ago

Thank you for sharing!

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Ann B
Ann B10 months ago

the government needs to spend more money on these important things and let the spending on space be GONE

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JT Smith
Past Member 10 months ago

The reason there hasn't been a cure is that there is zero profit in curing ANY disease; in fact, it's far more profitable to help people "live with" their ailments. As long as medicine is a business, profits will ALWAYS trump cures!

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Paulo R
Paulo R10 months ago

ty

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Paulo R
Paulo R10 months ago

ty

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Leo C
Leo C11 months ago

Thank you for sharing!

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Camilla Vaga
Camilla Vaga11 months ago

thanks

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Amanda M
Amanda McConnell11 months ago

thanks for sharing

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Amanda M
Amanda McConnell11 months ago

thanks for sharing

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Winn A
Winn A11 months ago

I really hope this new research leeds to advancements in the treatment of this disease.

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