by 
- Researchers are reporting that two new treatments can effectively target cholesterol at the genetic level.
- Both treatments are aimed at people with a genetic predisposition to high cholesterol for whom diet and exercise are not effective.
- Experts note that both treatments need more research and will likely take years to be approved.
Two promising new clinical trials looking at the genetics of cholesterol were revealed this week at the American Heart Association’s
Both drugs, designed to reduce harmful cholesterol levels, have been shown to be effective and safe, their creators said.
The drugs target people born with a genetic predisposition to high cholesterol, increasing the chances of heart attacks and strokes.
Current tools for managing high cholesterol include exercise, diet, and statin medications. These two new drugs would be the first to attack the genetic cause of high cholesterol.
However, neither will hit the market anytime soon, as both will require years of more research before they can be approved for consumers.
However, experts say the results are encouraging.
“The new studies… are truly a groundbreaking change in the world of cardiovascular medicine,” said Dr. Spencer Kroll, a physician at Kroll Medical Group and the Cholesterol Treatment Center in New Jersey and director of the Northeast Lipid Association.
“Even though they are in the early phase of clinical trials, these studies represent that controlling cholesterol and treating cardiovascular disease is a multimodal and individualized process,” said Kroll, who was not involved in the research. Medical News Today. “We are now entering the realm of cholesterol therapy, which moves away from one-size-fits-all statin treatment to personalized therapies for each patient.”
It is administered intravenously and targets the PCSK9 gene, which is associated with higher levels of LDL, also known as “bad cholesterol.”
The researchers explain that the drug makes a small change in PCSK9, limiting its ability to raise cholesterol. Theoretically, onceThe treatment is expected to last a lifetime, although so far test subjects have only been followed for six months.
The initial study had just 10 participants and was intended to evaluate the safety of the drug. Most participants did not receive doses large enough to make a measurable difference in their cholesterol levels, but made it through the trial without major side effects or health problems.
Researchers at Verve Therapeutic report that they administered higher doses to three individuals, whose LDL levels were reduced by more than half.
All study participants had a genetic disease called
“Although this first-in-human trial was very small, it showed a large drop in LDL levels in some patients,” said Dr. Cheng-Han Chen, interventional cardiologist and medical director of the Structural Heart Program at MemorialCare Saddleback. Medical Center in California that was not involved in the research.
“Most importantly, the study was successful in showing that the “base editing” technique can work in the liver of humans,” Chen said. Medical News Today. “These are very early clinical results that show proof of concept in humans, but require much more research before they can be used on a broader scale.”
Research on this medicine has not yet been published in a peer-reviewed journal.
A second potentially innovative therapy aimed at lowering cholesterol has also been
This targets a type of protein associated with cholesterol called lipoprotein. People with high levels of lipoprotein(a) are at high risk of fat and cholesterol accumulation in the arteries. Lipoprotein sticks to LDL cholesterol, potentially causing plaque buildup.
The condition is genetic and researchers have noted that exercise, diet and statins have little impact on lipoprotein levels.
The research team, which was funded by pharmaceutical company Eli Lilly, used a drug called lepodisiran, which helps block the production of a key protein component of the lipoprotein(a) particle.
The second study was also small – just 48 adults in the United States and Singapore with high lipoprotein(a) levels.
The researchers reported that the drug was considered safe, with no major side effects. They said the drug also reduced lipoprotein(a) levels by 94% over a year with a single dose.
Researchers noted that 64 million adults in the United States have high levels of lipoproteins.
O
Experts who were not involved in the research said Medical News Today that although the study results are promising, both treatments still have a long way to go.
“While this small proof-of-concept study of VERVE-101 is highly interesting, I presume that many hundreds if not thousands of similar patients would need to be treated to show the safety and effectiveness of this therapy before the Food and Drug Administration or other international regulatory bodies would consider approval,” said Dr. Wesley Milks, a cardiologist specializing in cardiovascular disease prevention, including treatment of lipid disorders, at Ohio State University Wexner Medical Center.
Milks said there are potential downsides to treating people at the genetic level.
“With an intervention that results in theoretically permanent editing of DNA, the irreversibility of such a therapy may be discouraging for many patients and healthcare professionals,” he noted. “I would certainly want to make sure that off-target genetic disorders or other undesirable genetic disorders would be extremely unlikely as a result of receiving this CRISPR gene editing therapy before recommending it to my own patients.”
What’s more, the treatments, when finally approved, could have other drawbacks, said Dr. Rekha Kumar, chief medical officer of the Found weight-loss program and former medical director of the American Board of Obesity Medicine.
“This is new because of the mechanism of action of these two potential new drugs, but both are far from everyday use,” Kumar said. “Both target specific genetic abnormalities related to cholesterol metabolism, making them a significant development as we move toward personalized therapies for cholesterol metabolism disorders.”
“When medicines are so personalized, they become very expensive, so we are talking about a high cost for a medicine that treats a specific population”, he added. “Often, many people are unaware that they have these disorders or do not have access to adequate care to be tested for these genetic abnormalities.”
