In two studies, experimental drugs for cholesterol show ‘revolutionary’ promise

The NBC News article discusses a groundbreaking drug by Verve Therapeutics, using gene-editing to target the PCSK9 gene, crucial in LDL cholesterol production. This innovative approach showed significant reductions in LDL cholesterol in early trials. Designed for one-time use, its long-term effects are still under study, focusing on individuals with genetically high cholesterol levels from birth.

A second drug, lepodisiran, targets the harmful cholesterol type, lipoprotein(a) or Lp(a), which is resistant to lifestyle changes. This drug works by inhibiting the body's production of Lp(a) through mRNA targeting. Early results show a dramatic reduction in Lp(a) levels, offering a new treatment avenue for this previously untreatable condition, potentially acting as an annual treatment.

These treatments mark a significant advancement in cholesterol management, especially for those with genetic risks. While promising in reducing heart attack and stroke risks, further research is needed to establish their long-term safety and efficacy. The medical community views these therapies as potential breakthroughs in cardiovascular disease treatment, pending further studies and regulatory approval.