A new treatment shows promise in removing cholesterol and a discovery that flips Alzheimer’s research on its head.
CLEVELAND —
A single shot at preventing heart disease
A single infusion. A dramatic drop in bad cholesterol. And results that may last a lifetime. An experimental gene-editing treatment is turning heads in the medical world — and researchers say it could one day change the way heart disease is prevented for millions of Americans.
The treatment, published in The New England Journal of Medicine, targets a gene in the liver called PCSK9. A tiny molecular machine, wrapped in a fat coating so it can travel through the bloodstream and slip inside liver cells, crawls along the cell’s DNA until it finds that gene. Then it erases a single DNA letter and replaces it with another. That one small change disables PCSK9, preventing the liver from making the protein it produces. Without that protein, the liver pulls more LDL cholesterol — the harmful kind — out of the bloodstream, keeping levels lower.
In a preliminary trial of 35 patients, all of whom had genetically high LDL levels or existing heart disease, the highest dose of the treatment reduced LDL by as much as 62%. In patients treated 18 months ago, those reductions appear to hold. A larger study of 200 patients is planned.
Cardiovascular disease kills nearly 800,000 Americans a year. Existing treatments — daily statin pills, injected PCSK9-blocking drugs — work well, but adherence is a persistent problem. Between one-third and one-half of patients stop taking cholesterol-lowering medications within a year of starting them, even after a heart attack. A one-time treatment could sidestep that barrier entirely.
High-cost gene therapies are typically developed for rare diseases — but the company behind this treatment, now a subsidiary of Eli Lilly, says affordability is a priority if the drug eventually reaches approval. The goal, researchers say, is a medicine that someday becomes part of routine primary care.
Still, experts urge caution. The Food and Drug Administration requires all gene therapy patients be followed for 15 years, and researchers acknowledge the need for significantly more safety data before the treatment could move toward widespread use.
New discovery could rewrite Alzheimer’s playbook
Researchers may be about to change the way scientists understand Alzheimer’s disease, and it starts with a gas your body already makes.
A new study from University Hospitals and Case Western Reserve University at Harrington Discovery Institute found that nitric oxide plays a broad role in regulating alternative splicing — the process by which a single gene gets edited into multiple different versions, dramatically increasing how many instructions our DNA can produce. Researchers found that nitric oxide levels are lower in Alzheimer’s patients’ brains, and that loss of this regulation correlates with greater plaque buildup and faster memory decline.
That finding directly challenges decades of scientific thinking. The field long believed nitric oxide levels were too high in Alzheimer’s brains and contributed to the disease. This study says the opposite is true.
The team also identified specific enzymes that strip nitric oxide from brain proteins involved in gene splicing — creating a nitric oxide-deficient state. That opens a potential new treatment target: blocking those enzymes to restore nitric oxide levels and potentially slow or treat the disease. Next steps include animal studies using a new class of enzyme inhibitors. The findings appear in the journal Molecular Cell.
Think before you supplement
Social media is loaded with supplement recommendations — but Cleveland Clinic says to treat them like medication. That means confirming you actually need one before you take it.
Taking too many supplements can strain the kidneys and liver and interact with prescription drugs. Many products lack scientific backing and fall outside Food and Drug Administration oversight. When shopping, look for third-party-tested products screened for heavy metals.
Pregnant women should use extra caution: certain supplements can harm a developing baby. Talk to your provider before adding anything new.