High cholesterol: New drug promises to ‘do what statins couldn’t’

Why cholesterol is bad for you

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A new generation of cholesterol lowering drugs and therapies are approaching the market, according to an article published by the American Chemical Society.

A mixture of oral drugs, injections and single dose gene therapies could cut cholesterol levels by more than half for a large number of at risk people.

One treatment, which promises a 60 percent reduction with only one dose needed, is set to begin human testing this year.

The drugs are based on research of a gene called PCSK9.

People who have a non-functional PCSK9 gene have naturally low levels of unhealthy LDL cholesterol.

The gene codes for a protein that discourages the uptake of LDL by cells, so people with an inactive gene clear more LDL from the blood.

The new therapeutics are looking to inhibit this gene or block the protein it codes for.

Attempts have been made previously to target the PCSK9 gene to lower cholesterol.

These proved ineffective in 2015 due to high cost and the need for new injections at two week intervals.

The new options claim to have fixed these problems, offering cheaper treatments that remain effective for longer.

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The treatments under development target the gene in different ways.

At the end of last year the Food and Drugs Administration gave approval for an RNA therapy that blocks the gene from being transcribed with a twice yearly injection.

Another blocks the protein with an oral pill.

One hopeful therapy aims to achieve a permanent effect with a single dose of CRISPR therapy, having already successfully achieved cholesterol reduction in monkeys.

CRISPR is a gene editing tool that allows for targeted removal or alteration of genes.

It uses a protein called Cas9 produced by bacteria that can be modified to target specific sequences of DNA and make cuts in it.

The technology is still in its infancy but achieved the Nobel Prize in Chemistry in 2020.

There are incentive structures in the medical industry that incentivise the production of statin alternatives.

Many statins are out of patent and can be produced and distributed at minimal cost.

Research in the  Medical Journal of Australia noted that use of generic statins over patented treatments confers savings of several billion pounds.

It may be good to discuss with your doctor what the best treatment is if you are concerned about your cholesterol levels.

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