study<\/a>, 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 \u2014 the bad kind \u2014 or heart disease.<\/p>\nIn the 35 patients, a single infusion of the highest dose of the treatment reduced LDL cholesterol levels by as much as 62 percent. The change has been sustained in a subgroup whose members were treated 18 months ago.<\/p>\n
It will be followed by a larger study of 200 patients.<\/p>\n
It is unusual for The New England Journal of Medicine to publish such a preliminary result. But \u201cit looks like it works pretty well,\u201d 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.<\/p>\n
Still, \u201cwe need much more safety data,\u201d said Dr. J. Michael Gaziano, the director of preventive cardiology at the Boston V.A. health care system, who was not involved in the new study. The Food and Drug Administration requires that all patients in gene therapy studies be followed for 15 years.<\/p>\n
Patients in the trial received an infusion containing a gene editing \u201cmachine,\u201d or a tiny molecular factory wrapped in a cloak of fat. The fat-coated particles travel through the blood directly to the liver, where they are taken up by cells that remove the fatty wrapper.<\/p>\n
The editing machine then crawls along the liver cell\u2019s DNA until it finds its target, a gene called PCSK9. It stops there and erases one DNA letter in the gene, replacing it with another.<\/p>\n
That simple change disables the PCSK9 gene and prevents cells from making the PCSK9 protein. Without it, the liver pulls more LDL cholesterol out of the bloodstream, keeping the levels lower.<\/p>\n
The study was led by Dr. Sekar Kathiresan, the chief executive at Verve Therapeutics, now a subsidiary of Eli Lilly. Dr. Kathiresan, a cardiologist, said he was motivated by personal history.<\/p>\n
His grandmother, father, uncle and brother all had heart attacks. His brother died of cardiac arrest at age 42, just after returning from a run.<\/p>\n
Gene therapies for rare diseases carry multimillion-dollar price tags. But Dr. Daniel Skovronsky, chief scientist at Eli Lilly, said that would not be the case if this treatment were eventually approved.<\/p>\n
\u201cThat\u2019s not what we\u2019re going for here,\u201d he said. \u201cWe\u2019re going for a medicine that someday could be part of primary care.\u201d<\/p>\n
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.<\/p>\n
But too many people cannot or will not take the drugs. Between one third and a half of patients stop taking cholesterol-lowering medications within a year of starting them, even people who have had heart attacks.<\/p>\n
Kristy Faulkner, 45, who lives in Guilford, Conn., is among those who need treatment but are reluctant to take a powerful drug. Heart disease runs in her family, and she had a heart attack when she was 42.<\/p>\n
\u201cThere\u2019s some sort of internal denial, like I can\u2019t be on these meds every day of my life,\u201d she said.<\/p>\n
\u201cI understand the importance,\u201d she added, \u201cand I feel ashamed.\u201d<\/p>\n
Her cardiologist, Dr. Erica Spatz, at Yale University, is hoping Ms. Faulkner\u2019s insurance will pay for a PCSK9 inhibitor that only has to be administered every six months. Her medical history means there is \u201cno margin for error,\u201d Dr. Spatz said.<\/p>\n
But a gene-editing treatment that is administered just once?<\/p>\n
\u201cThis kind of therapeutic breakthrough could be a game changer for people like her,\u201d Dr. Spatz said.<\/p>\n
Alice Thomas, 64, of Lexington, N.C., would love to take cholesterol-lowering drugs but can\u2019t get them. Her sole source of income is Social Security, and while statins are cheap, she was unable to tolerate them.<\/p>\n
Her insurance did not approve the injectable drugs that might have helped. She has had two strokes, and a few months ago, her LDL cholesterol level was dangerously high at 190.<\/p>\n
\u201cI didn\u2019t have anything,\u201d Ms. Thomas said. \u201cThen I found out about this study.\u201d<\/p>\n
She received the infusion in Dr. Kathiresan\u2019s trial on March 30. Two weeks later, her cholesterol level was 50.<\/p>\n
\u201cThis is great,\u201d she said. \u201cOne time and it\u2019s over.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"In a small, preliminary study, an experimental gene-editing treatment dramatically lowered cholesterol levels, perhaps permanently, after just one…\n","protected":false},"author":2,"featured_media":243834,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[6],"tags":[95235,95238,95236,95237,39989,172,95239,9,11,10,93289,3730],"class_list":["post-243833","post","type-post","status-publish","format-standard","has-post-thumbnail","category-new-york","tag-cholesterol","tag-eli-lilly-and-company","tag-genetic-engineering","tag-genetics-and-heredity","tag-heart","tag-medicine-and-health","tag-new-england-journal-of-medicine","tag-new-york","tag-new-york-headlines","tag-new-york-news","tag-preventive-medicine","tag-research"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts\/243833","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/comments?post=243833"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/posts\/243833\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/media\/243834"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/media?parent=243833"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/categories?post=243833"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us-ny\/wp-json\/wp\/v2\/tags?post=243833"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}