Beyond “One Shot Cures Heart Disease” Headline

A blockbuster study about a shot that alters the genes that control bad cholesterol production needs clarification.

Photo by digitale.de on Unsplash

One-and-Done Heart Disease Prevention? Scientists Show It May Be Possible.

This was the headline from the New York Times two days ago. This could be considered clickbait were it not for the question mark at the end of the first sentence and the word “possible” in the second. The answer is it is too soon to know. Is it promising? Yes. Is it a slam dunk? No.

The study was done in only 35 patients. All have genetically (inherited) high levels of LDL (bad) cholesterol. These patients are also known to have a much higher incidence of coronary artery disease (CAD) than the rest of the population. Other studies have shown that reducing LDL levels prevent adverse heart events, such as heart attacks and strokes.

There was a 62% reduction in LDL levels among the 35 patients. And it lasted for at least one year. This was after the VERVE company (now a subsidiary of Eli Lilly) developed a new, unique system to alter the gene that controls LDL production. And it does so after only one infusion. Hence, the headlines.

However, this was only a phase one study. (The FDA generally requires four phases before any drug or therapy can be submitted for approval.) Although the results are encouraging, here are the relevant contexts and considerations.

• Only 35 (soon to be only 200) patients were studied.
• These patients have an abnormal gene that produces unusually high levels of LDL (bad) cholesterol. Because of this, the therapy may or may not be applicable to other patients with only mildly to moderately elevated levels.
• It is unknown if this benefit will last more than one year. Importantly, “The Food and Drug Administration requires that all patients in gene therapy studies be followed for 15 years.”
• The 62% LDL reduction was observed only at the highest drug dosage, of which there were six. The number of patients treated is far too small to rule out serious long-term side effects.
• Although not specified, one can reasonably assume that the 62% figure is a relative, not an absolute, risk reduction. This difference is important. Read here to see why.

For many patients who can’t, or won’t, take LDL-lowering drugs, this could be a viable alternative—eventually. 15 years is a long time to wait for an answer. The good news is that other diseases might respond to this type of gene-altering technology, called CRISPR. Sickle-cell disease is currently being treated with this technology, and many more are likely to follow.

To summarize, this is exciting, but far from definitive, news. And unfortunately, it may be more than a decade before we have an answer and the treatment is approved.

I am a retired MD passionate about health, medicine, gardening, culture, and food.

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