Two new gene-editing treatments that target extremely high levels of cholesterol in people genetically predisposed to the condition were found safe and effective in two new studies. One of the treatments, from Verve Therapeutics based in Boston, targets the PCSK9 gene using a gene-editing approach. A tiny change is made to the gene, affecting the gene as an eraser, deleting the gene’s ability to fuel a rise in cholesterol levels. In Verve’s preliminary study, 10 patients received doses that did not make a measurable difference in their LDL levels, however 3 patients were given higher doses and their LDL levels decreased by more than half. Verve’s research was limited to people with the genetic disorder familial hypercholesterolemia. With this condition, cholesterol levels are high from birth and many patients suffer from heart attacks in their 30s or 40s. The second study demonstrated the possibility of treating high levels of lipoprotein(a) cholesterol. People with this genetic condition of high lipoprotein(a) are at a higher risk of having their arteries clogged because Lp(a) attaches itself to LDL making them stickier and more likely to cause plaque. The study involved 48 patients who were given a drug called lepodisiran, which targets mRNA. In this condition, mRNA tells the body to produce Lp(a) but the drug stops this process. Via the study, the drug was found to be safe with no major side effects but also dramatically lowered Lp(a) levels, decreasing levels by 94% for nearly one year.
The results presented by these two studies show really exciting, promising results. Even though there are still years of additional research before the FDA would consider approving them, these results show results that could be incredibly beneficial for individuals with high cholesterol levels. The first PCSK9 gene study could theoretically be a one-time treatment that would last an individual their lifetime. The mRNA gene study could also theoretically become a treatment similar to that of a yearly vaccination to treat the disorder. The possibilities created from both these studies could relieve individuals with those disorders of the daily prescriptions and injections, and from the strict diet and exercise. And more importantly, the expected lifespan could increase by a lot with a decrease in other medical risks like expected heart attacks. While these studies on gene therapy have made a big impact on treatments for genetically disposed high cholesterol levels, it also paves the way for a broader look into therapeutic gene therapies for different cardiovascular diseases.https://www.cdc.gov/genomics/disease/fh/FH.htm#:~:text=Familial%20hypercholesterolemia%20(FH)%20is%20a,sometimes%20called%20%E2%80%9Cbad%20cholesterol.%E2%80%9D