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Ishita Roy

Breakthrough: Gene Editing Jab Dramatically Lowers Harmful Cholesterol Levels In First Trial

It may sound unreal, but by just altering one gene could one day help many people to permanently lower their dangerously high cholesterol and blood fat levels. This is the biggest takeaway from the latest research which had been unveiled at the American Heart Association's annual meeting in New Orleans. 

The early stage human trial tested a first of its kind CRISPR based therapy, and the results were way better and way more powerful than what the cardiologists had expected. 

<h2>What Is This Experimental Treatment All About?</h2>

The Phase 1 trial involved 15 adults, all with severely high levels of LDL cholesterol, or the <span>bad cholesterol</span>. They also had high triglycerides, some had both. The main goal of the experiment was not to prove whether the drug would improve health, but to confirm if it is safe enough to be tested in humans. 

Dr Steven Nissen of the Cleveland Clinic, one of the study investigators, admitted he never imagined such a treatment would be possible. “The results were pretty spectacular,” he said.

Researchers say it passed that initial test. Even more striking, a single infusion of the gene editing therapy reduced LDL (the “bad” cholesterol) and triglycerides by around 50 percent. That kind of drop could sharply reduce a patient’s lifetime risk of heart attack and stroke.

This treatment uses a gene editing tool called CRISPR. This tool can cut and alter DNA. This is thus able to target a single liver gene called ANGPTL#. While usually this gene boosts cholesterol levels by blocking the liver's ability to break down fats, some people may have naturally versions of this gene that do not work as strongly, and thus give them low cholesterol levels their entire lives. 

The experimental drug is designed to mimic that natural advantage by turning off the gene permanently. Unlike statins, which must be taken daily, this therapy aims to work after just one dose. The drug, CTX310, was developed by CRISPR Therapeutics, which also funded the trial.

The findings, which were simultaneously published in The New England Journal of Medicine, sparked a mix of enthusiasm and hesitation among heart experts.

Dr Karol Watson of UCLA called it an important proof of concept, but emphasized that permanent gene changes require long term safety data. “We already have safe, effective medications,” she said. “CRISPR must prove it’s both effective and safe over the long haul.”

Others note that many patients stop taking statins due to side effects, meaning a one time alternative could eventually fill a real need. Still, doctors stress that such a therapy must be studied for years before it becomes a mainstream option.

Most participants in the study were in their 50s or 60s, and lived in Australia, New Zealand, and in the UK. They received a single IV infusion lasting up to four and four and a half hours. Side effects were mild and temporary: nausea, back pain, and one brief increase in liver enzymes. One participant died months later from an unrelated cause.

Those who received the highest dose saw the biggest change. LDL levels fell by nearly 49 percent and triglycerides dropped by about 55 percent within two months.

Experts like Dr Elizabeth McNally of Northwestern say the dual effect on both LDL and triglycerides is promising, especially for people with very high triglycerides. Still, she cautioned that researchers must prove the therapy offers clear advantages over existing treatments.

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It may sound unreal, but by just altering one gene could one day help many people to permanently lower their dangerously high cholesterol and blood fat levels. This is the biggest takeaway from the latest research which had been unveiled at the American Heart Association's annual meeting in New Orleans. The early stage human trial tested a first of its kind CRISPR based therapy, and the results were way better and way more powerful than what the cardiologists had expected. What Is This Experimental Treatment All About?The Phase 1 trial involved 15 adults, all with severely high levels of LDL cholesterol, or the bad cholesterol. They also had high triglycerides, some had both. The main goal of the experiment was not to prove whether the drug would improve health, but to confirm if it is safe enough to be tested in humans. Dr Steven Nissen of the Cleveland Clinic, one of the study investigators, admitted he never imagined such a treatment would be possible. “The results were pretty spectacular,” he said.Researchers say it passed that initial test. Even more striking, a single infusion of the gene editing therapy reduced LDL (the “bad” cholesterol) and triglycerides by around 50 percent. That kind of drop could sharply reduce a patient’s lifetime risk of heart attack and stroke.How Does Gene Editing Work?This treatment uses a gene editing tool called CRISPR. This tool can cut and alter DNA. This is thus able to target a single liver gene called ANGPTL#. While usually this gene boosts cholesterol levels by blocking the liver's ability to break down fats, some people may have naturally versions of this gene that do not work as strongly, and thus give them low cholesterol levels their entire lives. The experimental drug is designed to mimic that natural advantage by turning off the gene permanently. Unlike statins, which must be taken daily, this therapy aims to work after just one dose. The drug, CTX310, was developed by CRISPR Therapeutics, which also funded the trial.Excitement, But With CautionThe findings, which were simultaneously published in The New England Journal of Medicine, sparked a mix of enthusiasm and hesitation among heart experts.Dr Karol Watson of UCLA called it an important proof of concept, but emphasized that permanent gene changes require long term safety data. “We already have safe, effective medications,” she said. “CRISPR must prove it’s both effective and safe over the long haul.”Others note that many patients stop taking statins due to side effects, meaning a one time alternative could eventually fill a real need. Still, doctors stress that such a therapy must be studied for years before it becomes a mainstream option.How Was The Study Conducted?Most participants in the study were in their 50s or 60s, and lived in Australia, New Zealand, and in the UK. They received a single IV infusion lasting up to four and four and a half hours. Side effects were mild and temporary: nausea, back pain, and one brief increase in liver enzymes. One participant died months later from an unrelated cause.Those who received the highest dose saw the biggest change. LDL levels fell by nearly 49 percent and triglycerides dropped by about 55 percent within two months.Experts like Dr Elizabeth McNally of Northwestern say the dual effect on both LDL and triglycerides is promising, especially for people with very high triglycerides. Still, she cautioned that researchers must prove the therapy offers clear advantages over existing treatments.

breakthrough: gene editing jab dramatically lowers harmful cholesterol levels in first trial

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