One and done?
Pharma giant Eli Lilly announced Monday that its gene-editing therapy can reduce LDL (“bad”) cholesterol by up to 62% with a single infusion.
Not only was the experimental treatment successful, but the results were still apparent in some participants 18 months after they received the dose.
While most gene therapies target rare disorders, this new treatment could lower the risk of heart disease, the leading cause of death in the US.
A single dose of VERVE-102 “switches off” a gene known as PCSK9 that plays a role in the amount of LDL cholesterol the liver can detect and remove from the bloodstream. Less PCSK9 means less bad cholesterol.
An early-stage trial published this week in The New England Journal of Medicine tracked 35 patients with heart disease or genetically high levels of LDL cholesterol.
The highest dose of the medication — 1 milligram per kilogram of body mass — cut the PCSK9 protein in the blood by up to 88%. No serious adverse effects were reported.
“This drug turns off a tiny fraction of DNA, and your LDL cholesterol is lower by 50% for the rest of your life,” Riyaz Patel, an academic cardiologist at University College London, told Science Focus last year, before the study was peer-reviewed.
“That’s it,” Patel said. “One and done. This is going to be revolutionary.”
While the initial results are preliminary, a Phase 2 clinical study of VERVE-102 is expected to begin by the end of the year.
Existing medications that work the same way — by inhibiting PCSK9 production — have only reduced cholesterol between 50% and 60%.
Another benefit of the medication? LDL doesn’t creep back up the way it can with similar treatments.
Plus, VERVE-102 doesn’t need to be taken every day like traditional cholesterol-controlling statins.
“Your LDL-C is starting to revert back to normal before you get another hit,” Myles Minter, a healthcare analyst with William Blair, told Fierce Biotech.
“Every moment of LDL-C exposure matters for your lifetime risk of a cardiac event.”
High levels of LDL cholesterol increase the risk of plaque buildup in blood vessels, which can block blood flow to the heart.
Only time and further clinical trials will reveal the group of patients VERVE-102 best suits.
The innovative treatment could be particularly useful for younger patients who are predisposed to bad cholesterol.
A treatment that alters DNA for life may not be suitable for older patients, with researchers focusing on those with premature cardiovascular disease and genetically high cholesterol.
