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Revolutionary gene editing therapy cuts bad cholesterol by 62 percent with a single infusion that could last a lifetime
Early clinical trial results published in the New England Journal of Medicine show a gene editing therapy called Verve 102 reduced bad cholesterol by up to 62 percent in patients, with levels remaining low a year later. Doctors say patients may only need one infusion in their entire lifetime.
Early results from a groundbreaking clinical trial have shown that a gene editing therapy called Verve 102 can reduce levels of bad cholesterol by up to 62 percent with a single infusion, with those reductions sustained for at least a year. The findings, published in the New England Journal of Medicine, have been described by doctors as potentially revolutionary for the prevention of heart disease.
The trial involved 35 patients who either had inherited high cholesterol or had suffered a heart attack at a young age. All continued their regular medications but additionally received the gene editing therapy. Professor Riaz Patel, who works at the largest cardiac centre in the United Kingdom and supplied a third of the patients, said the results represent a first in medical history.
For the first time we are in a position where we can do this one and done approach, where you do not need to worry about someone's cholesterol levels in terms of risk of heart attacks because they will have had a treatment that will hopefully see them out for the rest of their life, Professor Patel explained. The therapy works by editing the gene for a protein called PCSK9, which normally interferes with the liver's ability to clear bad cholesterol from the blood.
Patient Jeanette, who had dangerously high cholesterol and whose father died from a stroke at age 68, described the treatment as life-changing. It has totally changed my life, I feel like I am a new person, she said after her cholesterol levels halved following the infusion. Another patient, Daniel, who had been in and out of hospital with chest pains, said the therapy was a game-changer.
While doctors stress it is still early days and future trial phases will include hundreds or thousands of participants, the results have generated genuine excitement about the prospect of preventing heart disease, the world's leading cause of death. Eight million people in the United Kingdom alone have heart disease and require cholesterol-lowering medicines. Doctors say it is too early to know whether patients could eventually stop taking statins altogether, but the potential implications of the therapy are enormous.