EDITED GENES MAY DELETE CHOLESTEROL!
TWO exciting geneediting therapies may offer new treatments for patients with genetic predispositions to high cholesterol!
Statins are highly effective for many with the arteryclogging condition, which puts people at risk for deadly heart disease and strokes. But as many as 29 percent of those prescribed the pills can’t take them due to distressing side effects — such as excruciating muscle pain.
However, scientists say the experimental treatment VERVE-101 could prove to be a viable alternative.
Dr. Sekar Kathiresan — one of the cardiologists who developed the onetime infusion — calls it a “potential game-changer.” VERVE-101 makes a tiny change to the PCSK9 gene — deleting its ability to raise cholesterol levels — and early study findings suggest high cholesterol could be reversed or prevented from becoming a problem at all! Dr. Andrew Bellinger — chief scientific officer of Verve Therapeutics, which spearheaded the research — explains, “Instead of daily pills or intermittent injections to lower bad cholesterol, this study reveals the potential for a new treatment option — a singlecourse therapy that may last decades.”
The team studied patients with the genetic condition familial hypercholesterolemia, which causes sky-high LDL — commonly called bad cholesterol.
Subjects given higher doses of VERVE-101 saw their LDL levels slashed by more than half!
Larger and longer-term studies are scheduled — but the therapy shows promise, experts say. Meanwhile, lepodisiran is being hailed as the firstever treatment to address lipoprotein(a), a particularly dangerous type of cholesterol, which is resistant to statins and affects 64 million Americans. Lp(a) is similar to LDL cholesterol but more sticky
— putting those who are genetically prone toward its development at extremely high risk of cardiovascular disease and stroke. Lepodisiran is designed to target cellular mRNAs, which tell the body to produce
Lp(a) — and shut down the process!
In a groundbreaking Cleveland Clinic study, a single shot of the drug safely slashed Lp(a) levels by more than 94 percent for almost a year — without major side effects.
Cleveland Clinic’s Dr.
Steven Nissen says further trials are planned and adds, “We’re working fast because there are patients dying every day from this previously untreatable disorder.”