Experimental gene-editing therapy keeps bad cholesterol down for a year

In a small study, a single infusion of an experimental gene-editing therapy reduced LDL cholesterol and triglycerides for a full year. The results offer an early sign that altering a gene in the liver may produce a durable effect on blood fats.

Among people given the highest dose, LDL cholesterol dropped by an average of 52.5% from the starting point, while triglycerides fell 47.8% after one year. The findings reflect changes in blood-fat measurements in a small group, not proof that the treatment lowers the risk of heart attacks or strokes.

The therapy, CTX310, uses CRISPR-Cas9 to target a liver gene called ANGPTL3. That gene produces a protein involved in controlling how the body processes circulating fats. When its activity is reduced, both LDL and triglyceride levels can fall.

The infusion is designed to carry the gene-editing tools into liver cells. Researchers then track the target protein and blood-fat levels to determine whether the intervention works as intended.

The follow-up study, published in The New England Journal of Medicine, included 15 participants who received different doses. Examining several dose levels in an early trial helps researchers gauge how strongly the treatment works and what safety concerns may appear.

Earlier results had shown reductions after about two months. The updated report asked whether those benefits would hold up over time, an important question for a therapy meant to act after just one infusion.

Long-term prevention also depends on recognizing risk; see research into a blood test aimed at preventing heart attacks and strokes.

No serious treatment-related adverse events were reported during the year of follow-up. Still, a study with only 15 people could miss rare problems, and one year is not long enough to reveal effects that develop slowly.

That concern is especially relevant for a treatment meant to make a lasting change. Researchers plan to keep monitoring participants for many years to see whether the effect remains and whether any delayed safety issues emerge.

CRISPR Therapeutics funded the study, and Cleveland Clinic said the lead author’s institution had received research support from the company. Larger trials will be needed to show how consistently people respond and how the treatment stacks up against existing ways to lower blood fats.

The durable changes give researchers reason to keep studying the approach. The key question ahead is whether a single infusion can safely reduce the health problems linked to high cholesterol and triglycerides in patients for whom the benefits justify a permanent genetic edit.

If you are interested in this topic, please read articles about Healthy eating for a happy heart: managing cholesterol levels through diet, and Walnuts could help reduce ‘bad’ blood cholesterol, high blood pressure.

For more information, please see articles about The Mediterranean diet could protect heart health in people with heart disease, and Omega-3 fatty acids and heart health: What you need to know.

Source: Cleveland Clinic.