High cholesterol levels can lead to heart disease, stroke, and other chronic ailments which mostly require a lifetime of medication for maintenance. It's costly and very limiting for individuals as someone who has hypercholesterolemia will have to restrict themselves from eating foods that increase levels of bad cholesterol. It's a great piece of news then, that a new clinical trial of a CRISPR-based treatment has been found to successfully cut cholesterol levels by up to 55 percent.
The trial involved ten people who had been determined to be genetically prone to hypercholesterolemia, by infusing the gene editing tools directly into the bloodstream, contrary to other typical CRISPR treatments, wherein scientists extract cells, edit them, and then reinsert them into the patient.
It may sound like a risky procedure, but Verve Therapeutics, the biotech company responsible for the trial, mitigated the risks of side effects by pairing the gene editing tools with nanoparticles. What this new treatment aims to do is to target the liver protein PCSK9 so that LDL levels can be maintained.
Although the results were astonishing, the trial was not without a hitch. One of the participants of the trial died from cardiac arrest. However, a review board indicated that the cause of death was likely due to underlying conditions.
Another patient suffered a heart attack, which might have been due to the treatment. But the patient had not disclosed having intermittent chest pains which would have excluded him from the trial. It's not completely perfect yet, but this first human trial bodes well for the future of CRISPR treatments of hypercholesterolemia, and possibly other conditions like sickle cell disease.
(Image credit: Scientific Animations/Wikimedia Commons)