Gene-silencing drug halves cholesterol levels with a single jab

[ScienceRocks]

Gene-silencing drug halves cholesterol levels with a single jab


3 hours ago

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(Credit: beawolf/Depositphotos)

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.

Gene-silencing drug halves cholesterol levels with a single jab

There's so many great things we could do with this awesome tech...I believe it is unmoral not to help people in need.

 

[Old Rocks]

"Immoral to not help people in need" LOL Mathew, you might take warning from what happened to the last person who went around stating things like that. It has been two thousand years, but there are still those that like to crucify people talking like that. And they have gained power in our nation now.

 

[TNHarley]

"Immoral to not help people in need" LOL Mathew, you might take warning from what happened to the last person who went around stating things like that. It has been two thousand years, but there are still those that like to crucify people talking like that. And they have gained power in our nation now.

you misspelled unmoral

 

[waltky]

Vaccine-like Drug Lowers LDL Cholesterol...

Vaccine-like Drug Lowers 'Bad' Cholesterol
June 19, 2017 | WASHINGTON — A drug that acts like a vaccine has been shown to lower "bad" cholesterol or LDL and reduce the amount of fatty plaque that can lead to heart attacks. The drug rallies the immune system against a molecule that contributes to atherosclerosis.

The drug, called AT04A, is a form of immunotherapy that targets an enzyme, called PCSK9, that contributes to the formation of harmful plaque in coronary arteries. In studies of mice fed fatty, Western-style diets putting them at risk for heart disease, researchers showed that AT04A reduced the total amount of cholesterol by 53 percent and shrank atherosclerotic plaques by 64 percent, according to Oliver Siegel, who is with the Austrian pharmaceutical company AFFiRiS. "More importantly in the mice, we have seen a very significant reduction in development of those plaques, said Siegel. "As a result, we believe the reduction of LDL which we have seen before would translate into a clinical benefit, i.e. reduction of plaque that would otherwise reduce inflammation that would otherwise be the cause of heart attack potentially." AT04A was able to rally the immune system to produce antibodies that blocked the PCSK9 enzyme in the circulation of mice during the study. The study was published in European Heart Journal.

The immunotherapeutic approach, according to Siegel, is similar to, but not a vaccine per se in the sense that it doesn’t target bacteria and viruses, just the harmful enzyme. In an accompanying editorial in the journal, Ulrich Laufs of Saarland University in Germany and Brian Ference of the University of Bristol noted that in people taking cholesterol-lowering drugs, including AT04A, there may be an increased risk of patients developing diabetes. But Siegel said nothing like that has cropped up so far in studies of the compound.

A selection of pastries, including doughnuts, bagels, rolls, croissants, turnovers and sticky buns are displayed in a New York coffee cart. In studies of mice fed fatty, Western-style diets putting them at risk for heart disease, researchers showed that AT04A reduced the total amount of cholesterol by 53 percent.​

The authors of the editorial wrote in the short term, having a long-acting, cholesterol lowering drug may far outweigh the slight risk of new onset diabetes. Currently, many people with high levels of cholesterol take daily medication that drives down "bad" LDL cholesterol; but, Siegel says compliance is less than ideal. "What a few studies have shown is that patients, who...have already had a heart attack, have a relatively low adherence or compliance rate and we would imagine that patients who didn’t have a heart attack would have an even lower compliance rate," said Siegel.

Fatty plaques that develop in the heart vessels can choke off the flow of blood, causing a heart attack. If a piece of plaque breaks off and travels to the brain, it can cause stroke. Diseases of the coronary arteries are a leading cause of illness and death worldwide. Siegel said the shot under the skin would have to be given once a year like an immunization. Early human clinical trials testing the compounds safety are nearly completed, and Siegel hopes to have a cholesterol-lowering, clot-busting drug on the market in the next few years.

Vaccine-like Drug Lowers 'Bad' Cholesterol

 

[badger2]

In the April 2017 issue of New England Journal of Medicine, there is

'This approach sequesters virtually all PCSK9 in the reticuloendothelial system , thus preventing it from binding to LDL receptors. Circulating PCSK9 is derived almost entirely from the liver....'
(Inclisiran in Patients with High Cardiovascular Risk with Elevated LDL Cholesterol, NEJM 376: 15)

Therefore, a cautionary abstract concerning hepatitis viruses:

HCV CD81 / PCSK9
PCSK9 and infection: a potentially useful or dangerous association? - PubMed - NCBI

 

[badger2]

PCSK9 is a serine protease, automatically linking prions and Alzheimer's. That is why we question the history of chronic wasting disease in Colorado: the deer were eating pine needles. Where is the forensic reports verifying this? Denver industrial pollution is the likely culprit in CWD. Did the forensic reports mention lichens in the stomachs of dead deer?

Lichen Serine Protease
Degradation of the disease-associated prion protein by a serine protease from lichens. - PubMed - NCBI

Anti-Prion Lichens
Lichens: unexpected anti-prion agents? - PubMed - NCBI

Alzheimer's / Prion Protein (see "4.3 Prion Protein")
Amyloid β-interacting partners in Alzheimer's disease: From accomplices to possible therapeutic targets. - PubMed - NCBI
'....triple helical nanotube structure....'

Lichens evolved a secretory apparatus to degrade foreign proteins. Soil manganese will degrade prions down to their 27kDa infectious core, prompting the question of efficacy in the sequestering of PCSK9 over the range of genomic expressions in patients with cardiovascular risk.