CRISPR cure for high cholesterol enters first human trial
A trial testing a new CRISPR-based treatment to lower cholesterol has officially kicked off in New Zealand. If it works as well as it did in animal trials, the one-and-done treatment could save countless lives — permanently lowering cholesterol and the risk of a heart attack.
Cholesterol is a waxy material produced by the liver and found in certain foods we eat. It circulates in the blood, and it comes in two varieties: HDL and LDL. Having a healthy amount of HDL can protect you against disease, but excess LDL is the primary cause of coronary heart disease — a leading cause of death globally.
Pills called statins can lower cholesterol, too, but they must be taken once a day and can have intolerable side effects. Newer injectable meds can be taken as infrequently as twice a year, but they’re costly and often not covered by insurance.
This new treatment is designed to permanently deactivate a gene in the liver that controls the production of PCSK9 — a protein that prevents the removal of excess cholesterol from the body. In monkey trials, it reduced LDL cholesterol levels by 70% in just two weeks and kept them low for at least two years — suggesting that it could effectively cure high cholesterol in people.
While CRISPR is sometimes used to cut DNA, Verve’s treatment just swaps out a single letter in the PCSK9 gene for another. This is called “base editing,” and in animal studies, it’s been more efficient and had fewer errors than standard CRISPR.
In this trial, approximately 40 adults with “heterozygous familial hypercholesterolemia” (HeFH), a relatively common genetic disorder that causes dangerously high cholesterol and heart disease, will involve. The trial is only just beginning, but if everything goes as hoped, Verve’s treatment to lower cholesterol could mark CRISPR’s biggest impact on humanity yet.
https://bigthink.com/health/crispr-cholesterol-gene-therapy
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