A new treatment promises to eliminate cholesterol, and the discovery revolutionizes Alzheimer’s research.
cleveland —
A dose of heart disease prevention
Single infusion. Bad cholesterol plummets. The results may last a lifetime. An experimental gene-editing therapy is raising eyebrows in the medical community — and researchers say it could one day change the way things are Heart Millions of Americans can prevent disease.
Treatment methods published in new england journal of medicinetargets a gene called PCSK9 in the liver. A tiny molecular machine is wrapped in a fatty coating so that it can enter the liver cell through the bloodstream and crawl along the cell’s DNA until it finds that gene. It then deletes one DNA letter and replaces it with another. This tiny change causes PCSK9 to become inactive, preventing the liver from making the proteins it produces. Without this protein, the liver absorbs more LDL cholesterol (bad cholesterol) from the blood, keeping levels low.
In a preliminary trial of 35 patients, all of whom had genetically high LDL levels or had heart disease, the highest dose of treatment reduced LDL by as much as 62%. These reductions appeared to persist among patients treated 18 months ago. A larger study of 200 patients is planned.
Cardiovascular disease kills nearly 800,000 Americans each year. Existing treatments—daily statins, injections of PCSK9-blocking drugs—work well, but compliance is a persistent problem. One-third to one-half of patients stop taking cholesterol-lowering drugs within a year of starting them, even after a heart attack. A one-time treatment can circumvent this hurdle altogether.
High-cost gene therapies are typically developed for rare diseases, but the company behind the therapy, now a subsidiary of Eli Lilly and Co., said affordability would be a top concern if the drug is ultimately approved. The researchers say the goal is to one day become part of routine primary care.
Still, experts urge caution. The U.S. Food and Drug Administration requires 15 years of follow-up for all patients who receive gene therapy, and researchers acknowledge that more safety data is needed before the treatment can be widely used.
New discovery could rewrite Alzheimer’s disease script
Researchers may be on the verge of changing the way scientists understand Alzheimer’s disease, which starts with a gas your body already produces.
one new research Research from University Hospitals and Case Western Reserve University’s Harrington Discovery Institute found that nitric oxide plays a broad role in regulating alternative splicing, a process in which a single gene is edited into many different versions, greatly increasing the number of instructions our DNA can produce. Researchers have found that people with Alzheimer’s disease have lower levels of nitric oxide in their brains, and the loss of this regulation is linked to increased plaque buildup and faster memory loss.
The discovery directly challenges decades of scientific thinking. The field has long believed nitric oxide levels are too high high Alzheimer’s disease in the brain and causes the disease. This study shows the opposite is true.
The team also discovered specific enzymes that remove nitric oxide from brain proteins involved in gene splicing, creating a nitric oxide-deficient state. This opens up a potential new therapeutic target: blocking these enzymes to restore nitric oxide levels and potentially slow or treat the disease. Next steps include animal studies using novel enzyme inhibitors. The research results appear in Journal of Molecular Cells.
Think twice before supplementing
Social media is awash with supplement suggestions, but the Cleveland Clinic says to treat them like medications. This means making sure you actually need it before taking it.
Taking too many supplements can stress your kidneys and liver and interact with prescription medications. Many products lack scientific support and are not regulated by the Food and Drug Administration. When shopping, look for products that have been third-party tested and screened for heavy metals.
Pregnant women should take extra caution: Some supplements may harm their developing baby. Please check with your provider before adding any new content.
