Researchers have completed a study that details the alleged success of a new gene-editing medicine that could help moderate genetically inherited high cholesterol in patients.
A single infusion of the medicine could help patients for decades.
Scientists presented their findings in November at the American Heart Association’s (AHA) “Scientific Sessions 2023” conference. The new gene-editing medication, called VERVE-101, works by “permanently” shutting down the PCSK9 gene in the liver. PCSK9 plays a role in regulating cholesterol in the blood.
The study — the first human trial of the medicine — included two women and seven men with an average age of 54 who had been diagnosed with heterozygous familial hypercholesterolemia. This inherited genetic disorder causes dangerously high cholesterol levels.
While some people are genetically predisposed to high cholesterol, most in the United States get high cholesterol from living unhealthy lifestyles. Having high levels of cholesterol, which is a type of fat in the bloodstream, can lead to artery blockage and heart issues. High cholesterol can be warded off through regular exercise and maintaining a healthy diet, as previously reported by The Dallas Express.
Each participant received a single intravenous infusion of the medication in dosages ranging from 0.1 mg/kg to 0.6 mg/kg. Researchers found that the medication successfully reduced cholesterol levels in the participants.
“The study found that the highest-two VERVE-101 doses: reduced LDL-C by 39% and 48% in the two participants receiving 0.45 mg/kg of the drug and 55% in the sole participant receiving 0.6 mg/kg; reduced blood PCSK9 protein levels by 47%, 59%, and 84% in the three participants receiving the 0.45 mg/kg or 0.6 mg/kg doses; [and] reduced LDL-C at six months in the sole participant receiving 0.6 mg/kg, with follow-up ongoing,” according to the AHA.
One participant had a heart attack, while another died as a result of cardiac arrest. However, these patients had underlying advanced coronary artery disease. “Most adverse events that occurred in the study were mild and unrelated to treatment,” said the AHA.
“All safety events were reviewed with the independent data safety monitoring board, who recommended continuation of trial enrollment with no protocol changes required,” said Andrew M. Bellinger, senior author of the study and chief scientific officer at Verve Therapeutics, according to the AHA.
Bellinger highlighted the difference such medicines could make in people’s lives.
“Instead of daily pills or intermittent injections over decades to lower bad cholesterol, this study reveals the potential for a new treatment option – a single-course therapy that may lead to deep LDL-C lowering for decades,” said Bellinger.
Sekar Kathiresan, Verve Therapeutics CEO and cofounder, plans to continue human trials. However, due to the incidents with the two patients who suffered cardiac complications, the organization intends to conduct tests on individuals who do not have advanced diseases or those at high risk for a heart attack, reported ScienceNews.
Scientists have long been trying to use gene editing tools and technology to solve medical problems and enhance patients’ lives. The FDA recently announced that it had approved two gene therapies based on CRISPR technology to treat sickle cell disease, as previously reported by The Dallas Express.
