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Gene-silencing drug halves cholesterol levels with a single jab

By Rich Haridy
March 20, 2017
Gene-silencing drug halves cholesterol levels with a single jab
A new gene-silencing drug gives hope of a future where keeping LDL cholesterol levels low is as simple as a small injection from your GP once or twice a year
A new gene-silencing drug gives hope of a future where keeping LDL cholesterol levels low is as simple as a small injection from your GP once or twice a year
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A new gene-silencing drug gives hope of a future where keeping LDL cholesterol levels low is as simple as a small injection from your GP once or twice a year
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A new gene-silencing drug gives hope of a future where keeping LDL cholesterol levels low is as simple as a small injection from your GP once or twice a year

A new gene-silencing drug that can lower cholesterol levels is proving promising in initial clinical test results. The treatment was shown to reduce the levels of low-density lipoprotein (LDL) cholesterol in patients' blood by up to 51 percent in the month following one single treatment. The drug, named Inclisiran, utilizes a technique called RNA interference therapy which targets, and switches off, a specific gene known to be responsible for elevated LDL levels.

A phase 2 clinical trial recruited 497 patients with high cholesterol, 73 percent of which were already taking statins, the current standard treatment for those at risk of cardiovascular disease.

Those patients who received a single dose found cholesterol levels reduced by on average 42 percent, six months following the dose. A group given two doses across three months found levels reduced up to 52 percent after six months. All the non-placebo groups in the study saw their cholesterol levels staying low for at least eight months following the treatment with no additional side effects.

"We appear to have found a versatile, easy-to-take, safe, treatment that provides sustained lowering of cholesterol levels and is therefore likely to reduce the risk of cardiovascular disease, heart attacks, and stroke," says lead author of the study, Professor Kausik Ray. "These reductions are over and above what can be already be achieved with statins alone or statins plus ezetemibe, another class of cholesterol-lowering drug."

A recently approved monoclonal antibody treatment called Evolocumab has also been finding notable success in reducing LDL cholesterol levels. Evolocumab has also had extraordinary success in broad trials, with LDL cholesterol reductions of up to 60 percent in patients undergoing that treatment.

The novel strength that Inclisiran is showing over Evolocumab is the minimal dosing regime. Ultimately, the authors of the Inclisiran study see the treatment as being as innocuous as visiting your GP once or twice a year for an injection, in much the same way as one would receive a flu vaccination.

"We believe that these clinical visits might only be twice a year at most, so ultimately, they are more convenient and more effective for patients and their health," explains Professor Ray.

The next stage of the trials will increase the volume of patients and asses the long-term safety of the treatment. The study notes that they are in the early stages of this clinical work, adding that much more research is needed before this drug will be publicly available. For those with cholesterol problems though, it seems there are a number of exciting new developments on the horizon.

The team's study is published in the New England Journal of Medicine.

Source: Imperial College London

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Medicalgene therapyHeart DiseaseRNACholesterol
8 comments
Rich Haridy
Rich Haridy
Rich has written for a number of online and print publications over the last decade while also acting as film critic for several radio broadcasters and podcasts. His interests focus on psychedelic science, new media, and science oddities. Rich completed his Masters degree in the Arts back in 2013 before joining New Atlas in 2016.

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8 comments
Rui Marques
I don't think that drugs like this should be available to population in general. We are reaching a point of dangerous carelessness just because the drugs exist to make us healthy no matter how we treat our body. This should be available for people with genetic problems and that's it. Diabetes, high cholesterol, etc. are diseases mostly caused by a total disrespect and disregard of the human body, of shortcuts on how we eat and live. Lots of sugar, fats and lack of exercise.
JimFox
Rui Marques Typically ill-informed. Most cholesterol is a natural product of the liver. external sources can have big effects but I, for example have always had high cholesterol- and weigh 70 kg. My liver over-produces it. I don't like fatty foods, dairy or red meat so rarely eat those. At 71, my BP is regularly 105/60, pulse 62. Explain THAT!
Cholesterol is often demonized in public press and medical literature for increasing the risk of atherosclerosis and heart disease. This simplistic view has fueled a number of myths and clouded the truth about the role of cholesterol in the body. Cholesterol is needed for the synthesis of sex hormones, adrenal hormones, bile acids and vitamin D. In fact, it is naturally produced in the liver. The liver makes about 75% of the cholesterol the body needs every day. The rest comes from the diet. Although, many physicians and health experts relentless advocate reducing blood cholesterol level, there is no scientific evidence to suggest that lowering total blood cholesterol reduces the risk of heart disease. In fact, a growing number of physicians report that many patients with heart disease have cholesterol levels well within normal ranges and that **many who tested with high cholesterol levels are quite healthy. There are generally 2 types of blood cholesterol. HDL (high-density lipoprotein) cholesterol and LDL (low-density lipoprotein) cholesterol. While HDL cholesterol is known as “good cholesterol” and LDL cholesterol is regarded as “bad cholesterol”, the truth is that cholesterol is neither good nor bad. Of more importance to cardiovascular health is triglycerides, another group of fats found in the blood. Studies show that measuring the amount of triglycerides in the blood is a better way to predict the risk of heart disease. More importantly, the ratio of triglycerides to HDL cholesterol is a pretty accurate predictor of cardiovascular health.
VirtualGathis
I'll have to add my vote that Rui needs to lay off the conspiracy theory shows and the hard core psychoactive drugs.
Diabetes has a huge mythos surrounding it, none of it true. Diabetes can be caused by environmental factors, typically due to toxins in the environment and diet, but is generally due to genetic predisposition. Diet and exercise can delay its onset, but not prevent it. They help in it's management, but do not eliminate the need for medication to manage insulin resistance.
On the subject of cholesterol, the same genes that cause high glocose (diabetes) also cause high cholesterol. IF they can silence this gene hopefully they can silence the insulin resistance gene as well.
Marilyn Harris
Serious side effects are very high at 11%!
http://www.nejm.org/doi/full/10.1056/NEJMoa1615758#t=articleResults
Safety
Adverse events were reported in 76% of the patients who received inclisiran and in 76% of the patients who received placebo (Table 3TABLE 3
Adverse Events That Occurred during Treatment through Day 210.). Most of these events (95%) were mild or moderate in severity (grade 1 or 2). The incidence of serious adverse events was 11% among patients who received inclisiran and 8% among patients who received placebo.
P51d007
It's a genetic thing in our family. In the late 60's, early 70's, my grandmother was told she had high levels...well, granted, she cooked everything in lard, salted everything, you know...old school cooking (sure tasted good!) Everyone in her family either died of heart attack or stroke. My dad went on low cholesterol diet & statins in the mid 70's and still has cholesterol levels in the mid to high range. I stayed low until my mid 40's then mine went up. Runs in the family. The only thing that bothers me is if there is something that can bring it down at a genetic level, what ramifications could that have down the road with reproduction? When you start screwing with the DNA, you could open up a can that you won't be able to put the lid back on.
Douglas Bennett Rogers
High blood cholesterol and LDL is a symptom of autoimmune disease caused by foreign bodies in the blood, largely gluten fragments. It served to keep primitive people from bleeding to death.
habakak
...This should be available for people with genetic problems and that's it...
JimFox, Rui clearly stated that people like you should have access to drugs like these. His other points are valid. Most of the people I know that are using medication are overweight and out of shape. And none of the people I know who exercise and watch their weight are using any drugs to control cholesterol, sugar or blood pressure.
Maybe you and others do not like the way he brings his point across, but he has a valid point. As a society we are overweight and inactive and that DOES lead to most of the issues people have with blood pressure, cholesterol and sugar.
Martin Winlow
Habakak is quite rught to support Rui's comment. We are only just scratching the surface of our understanding of how our bodies work and simply trying to use drugs to replace sensible diet and exercise to combat these extremely common but very modern diseases is extremely narrow-minded and silly. I would urge you all to research 'Prof John Rudkin' and his '70s work on this subject. No conspiracy 'theory', just depressing truth.