Researchers have made an exciting breakthrough with a new drug that targets lipoprotein(a), a cholesterol-like particle linked to heart attacks and strokes. Many people don’t know their Lp(a) levels could be high because lifestyle changes can’t affect it. The Cleveland Clinic has referred to Lp(a) as “one of the last untreatable frontiers of cardiovascular risk.”

The experimental drug, called lepodisiran, is designed to deactivate the gene that produces Lp(a). According to the Cleveland Clinic, there are other gene therapies in development as well. The findings were published in The New England Journal of Medicine and shared at the American College of Cardiology meeting on March 30.
Lp(a) levels are elevated in about 20-25% of the global population, which means around 64 million people in the U.S. and 1.4 billion people worldwide could be at risk. Unlike LDL cholesterol, which is influenced by lifestyle, Lp(a) is heavily determined by genetics. This was emphasized by Dr. Deepak L. Bhatt of Mount Sinai Fuster Heart Hospital, who explained that having high Lp(a) is inherited.
Dr. Steven Nissen from the Cleveland Clinic noted that while lifestyle factors can lower LDL cholesterol levels, they do not affect Lp(a). Currently, no approved medications can lower Lp(a), making lepodisiran a potential game-changer.
In a recent clinical trial involving 320 participants from various countries, those receiving lepodisiran showed a nearly complete reduction in Lp(a) levels after just one dose. Many maintained this reduction for at least a year after a second dose. Cardiologists are optimistic that this treatment could help millions of Americans and reduce cardiovascular risks.
While the results are promising, there were some limitations. A small percentage of participants experienced mild side effects at the injection site. The study also included few Black participants, a group more likely to have high Lp(a) levels, so more research is needed there. The study didn’t prove that lowering Lp(a) levels leads directly to fewer heart attacks or strokes, something further trials will aim to clarify.
Notably, experts recommend that adults check their Lp(a) levels at least once in their lifetime. Dr. Nissen pointed out that Lp(a) levels remain consistent throughout life, underscoring its genetic nature. As Ben Hwang, a doctor specializing in cardiovascular health, stated, “Knowing your Lp(a) levels can help you take preventive measures early on.”
For more detailed information on cardiovascular health, you can refer to the American Heart Association here.
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