A One-Time Treatment for High Cholesterol Shows Promising Results

The fact that the people in the study were able to maintain lower levels of LDL and triglycerides for up to a year means that CRISPR was able to edit enough cells in the liver, where much of the body’s cholesterol and triglycerides are produced, to give patients the benefit of having lower levels of the lipids. It also shows that these edited cells continue to produce new generations of cells that carry the CRISPR edit in AGNPTL3. People who received the highest dose of the CRISPR therapy saw levels of the ANGPTL3 enzyme drop by nearly 80%, which contributed to a drop in LDL and triglycerides of about 50%. “This is a really big step for CRISPR to show the durability of the result,” says Dr. Luke Laffin, co-director of the Center for Blood Pressure Disorders at the Cleveland Clinic and lead author of the study.

The CRISPR therapy did not cause significant side effects—and researchers did not expect it to, since people born with the defective ANGPTL3 gene don’t seem to have serious diseases either. That means the one-time therapy could potentially replace the current treatment for high LDL and triglycerides: daily statin pills. While effective, many people don’t take the pills on a daily basis for years, which lowers their effectiveness. “We could look at a situation further down the line where we are able to give people a choice,” says Laffin. In the future, if someone has a serious family history of heart disease or high cholesterol, for example, “this therapy may be an option for them where we could treat them now, edit their [liver cells], and they can continue to have a genetic defect that we know is safe and will control their cholesterol.”