A one-time injection of an experimental gene therapy from CRISPR Therapeutics showed that it was safe and reduced levels of harmful cholesterol and harmful triglycerides by half in 4 people who took the highest dose, and this raises hope for a single-dose treatment.
“We didn’t have anything that reduced both bad cholesterol and triglycerides by about 50%,” said Steven Nissen, a cardiologist at the Cleveland Clinic in the United States, who is the lead researcher in the first study of the treatment in humans.
Doctor Luc Lavin of the Cleveland Clinic, a researcher involved in the study, said: Although the development of the treatment is still at a very early stage, if future experiments prove that the treatment “CTX 310” is safe and effective, this may change medical practice.
He continued: “Instead of taking pills once a day or taking monthly injections, this treatment could potentially provide a safe and permanent one-time injection for patients with high cholesterol.”
The results were presented yesterday, Saturday, at the American Heart Association meeting in New Orleans and published in the New England Journal of Medicine.
High low-density lipoprotein, or so-called “bad” cholesterol, may cause deposits to accumulate in the walls of the arteries, which increases the risk of a heart attack or stroke. High triglycerides, another fat in the blood, may also increase these risks.
Turn off the gene
CTX 310 works by turning off a gene called ANGBTL3 through a single two-hour injection. It is inspired by studies showing that those born with an inactive copy of the ANGBTL3 gene have a lower lifetime risk of heart disease with no obvious negative consequences.
Regeneron’s Evkiza drug, which treats a rare genetic disorder, targets the same gene, but requires monthly injections.
The CRISPR trial, which was conducted in Australia, New Zealand, and the United Kingdom, participated in 15 patients between the ages of 31 and 68 years, and 5 different doses were tested on them. All participants had high triglycerides, high LDL cholesterol, or both, and failed to respond to other treatments.
Among the four patients who received the highest dose, triglycerides fell on average by 55% and LDL cholesterol by 50% after two weeks of treatment. Levels remained low for at least two months.
“We will try to prove the safety and effectiveness of these one-time treatments because we believe these options are important for patients,” Nissen said.
Three participants had temporary reactions to the treatment, including nausea and elevated liver enzymes that quickly resolved, Lavin said.
Participants will be monitored for a year after the trial, with the option of following up for an additional 15 years.