One-Shot Treatment Permanently Lowers Bad Cholesterol in Trials

Cholesterol comes in many forms, and LDL is the one considered bad, a major cause of arteries clogging up leading to heart attacks. The treatment for many decades now has been a class of medicines called statins. They have proven very effective at lowering cholesterol levels. Statins have to be taken daily and often forever. But gene therapy has shown that a one-and-done treatment to permanently lower LDL levels is not just possible but imminent.

A paper published in The New England Journal of Medicine on May 25 revealed that a phase 1 study by Eli Lilly administered an infusion of VERVE-102, a gene therapy, on 35 people with a history of premature coronary artery disease or a gene defect that increased cholesterol levels. The results were startling: LDL levels were reduced by 62 percent. And it remained at low levels when they did follow-ups for over a year. A protein called PCSK9 is linked to more LDL in the blood because it removes the receptors that clear LDL. The therapy turned off the gene that produced the protein, which meant that there were now more receptors to clear the LDL.

Such efficacy has significant implications for moving on to further trials, which will have larger sample sizes and longer-term follow-ups for efficacy and side effects. Gene therapy has already proven useful as a treatment, but to solve a metabolic disorder with just one dose could mean billions of human beings could eventually benefit. This is especially true for India. Studies have estimated that more than a quarter of the Indian population has elevated cholesterol levels. Heart disease ranks as the biggest killer in the country.

However, a gene therapy like this will take its time before becoming available. And even then, it will be exorbitantly priced in the beginning because it involves only one dosage.