Researchers are aiming to use a single gene therapy treatment to reduce the risk of heart attacks in vulnerable patients, according to The Guardian.
The therapy is being developed by a startup, called Verve Therapeutics, founded by a former Harvard medical school cardiologist and director of the Center for Genomic Medicine at Massachusetts general hospital, Sekar Kathiresan.
After spending 15 years identifying the genes linked to heart attack risk, he founded the startup, which has received $58.5 million from investors including a venture fund from Alphabet, Google’s parent company.
According to Kathiresan:
“The most exciting part of this is changing from a chronic care approach to a ‘one and done’ approach. We really think we can turn the tide against coronary disease by moving from a chronic care model to a one-time treatment.”
The treatment aims to use Crispr-Cas9 gene editing to switch off genes that lead to elevated levels of low-density lipoprotein (LDL), a form of cholesterol that builds up in the bloodstream and can block arteries.
The one-time injection contains nanolipids, which are tiny, fatty particles that target liver cells. They release Crispr-Cas9, which finds and turns off a gene called PCSK9. Switching off 30 to 40 percent of the gene has effects similar to natural mutations that protect from heart attacks. One, found in one in 50 African Americans, results in just one instead of two copies of PCSK9.
Those with the mutation are “healthy and remarkably resistant to heart attacks,” Kathiresan says.
A University of Pennsylvania geneticist, Kiran Musunru, has used gene editing to modify PCSK9 in mice, and succeeded in reducing cholesterol by 35 to 40 percent.
Heart disease is the top cause of death in much of the world, with 18 million deaths annually, 85 percent of which are caused by heart attacks and strokes. Patients at risk for heart attacks take statins to lower cholesterol, drugs for high blood pressure, and blood thinners. Doctors estimate that 80 percent of heart attacks can be prevented with a healthy lifestyle.
The first patients in the trial will be a group with a rare genetic disorder, called homozygous familial hypercholesterolaemia (FH). The condition is life-threatening, since most with the condition have heart attacks in their 30s or 40s. As a result, the potential benefits of the trial outweigh the risks that come with any experimental therapy.
If proven safe and effective for this group, further trials will offer the shot to wider populations.