紐時賞析／一次性預防心臟病 有望成真

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

一次性預防心臟病 有望成真

In a small, preliminary study, an experimental gene-editing treatment dramatically lowered cholesterol levels, perhaps permanently, after just one infusion, scientists reported Monday.

科學家周一表示，一項小規模初步研究顯示，一種實驗性基因編輯療法只需輸注一次，就能大幅降低膽固醇，而且可能終身有效。

If confirmed in larger studies, researchers hope the findings may lead to a one-and-done way to prevent heart disease in large numbers of people. Most gene therapies target rare diseases, but cardiovascular disease kills nearly 800,000 Americans a year.

如果後續更大型研究能證實這項結果，研究人員希望這種作法未來可能成為一次到位、用來預防大量民眾心臟病的方法。大多數基因療法原本是用來治療罕見疾病，但心血管疾病每年奪走近 80萬名美國人的生命。

“We have these debates and new guidelines that we should be treating people earlier,” said Dr. John H. P. Alexander, a cardiologist at Duke University who was not involved with the study. “A curative therapy would change the game.”

未參與研究的杜克大學心臟科醫師亞歷山大說：「我們一直在討論，也有新的指引，認為應該更早治療患者。如果有一種能根治的療法，情勢就會完全改變。」

The study, published in The New England Journal of Medicine, was an interim analysis of 35 patients in a trial that will involve as many as 85 participants. All have genetically high levels of LDL cholesterol—the bad kind—or heart disease.

研究刊登於《新英格蘭醫學期刊》，是針對一項臨床試驗中35名患者所作的期中分析；該試驗最多將納入85名參與者。所有患者都有遺傳性低密度脂蛋白膽固醇，也就是「壞膽固醇」偏高的問題，或患有心臟病。

In the 35 patients, a single infusion of the highest dose of the treatment reduced LDL cholesterol levels by as much as 62%. The change has been sustained in a subgroup whose members were treated 18 months ago.

在這35名患者中，接受最高劑量療法單次輸注後，低密度脂蛋白膽固醇最多下降62%。其中一個小組的患者在18個月前接受治療，效果至今仍維持穩定。

It will be followed by a larger study of 200 patients.It is unusual for The New England Journal of Medicine to publish such a preliminary result. But “it looks like it works pretty well,” said Dr. Eric Rubin, the editor-in-chief. Moreover, he noted, the trial is an ambitious attempt to apply cutting-edge gene therapy to the leading cause of death in the United States.

接下來，研究團隊將進行一項規模更大的試驗，納入200名患者。《新英格蘭醫學期刊》通常不太會刊登這麼早期的研究結果。不過總編輯魯賓表示：「看起來效果相當不錯。」他還指出，這項試驗是一次相當大膽的嘗試，試圖把尖端基因療法用在美國人頭號死因上。

High LDL levels are eminently treatable with an array of medicines, including the old standbys, daily statin pills. More recent advances include injected drugs that block the protein made by the PCSK9 gene, creating the same effect as gene editing.But too many people cannot or will not take the drugs. Between one-third and one-half of patients stop taking cholesterol-lowering medications within a year of starting them, even people who have had heart attacks.

低密度脂蛋白膽固醇偏高其實有很多藥物可治療，包括老牌的每日口服他汀類藥丸。近年較新進展，包括可阻斷PCSK9基因所製造蛋白質的注射型藥物，能產生與基因編輯相同的效果。但問題是，很多人不是不能吃，就是不願意吃。即使是曾經心肌梗塞的患者，也有三分之一到一半的人，在開始服用降膽固醇藥物一年內就停藥。

文／Gina Kolata，譯／國際中心