VERVE-102と呼ばれる実験的な治療法が、初期の安全性試験において1回の投与でLDLコレステロールを最大62％低下させた。この結果は、高コレステロール血症または初期の心血管疾患を有する患者35名を対象とした第I相臨床試験で得られたものである。データは今週、『ニューイングランド・ジャーナル・オブ・メディシン』誌に掲載された。
研究チームによると、この治療法は最高用量においても深刻な副作用は見られず、安全であると報告されている。被験者には肝酵素の一時的な軽微な上昇が見られたのみであった。この薬はナノ粒子に封入されたmRNAを用いて肝細胞内のPCSK9遺伝子を編集し、LDL値を上昇させるタンパク質の生成を恒久的に抑制する仕組みとなっている。
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Scientists at UT Southwestern Medical Center report they have identified a protein, HELZ2, that acts as a key regulator of how many cholesterol-carrying particles the liver releases into the bloodstream by affecting the gene APOB. The study was published in the American Heart Association journal Circulation and could inform future research into heart disease and fatty liver disease.
An experimental oral pill called enlicitide lowered LDL cholesterol by about 60% in a large phase three clinical trial, according to results published in The New England Journal of Medicine. The trial, led by Dr. Ann Marie Navar at UT Southwestern Medical Center and sponsored by Merck, involved 2,909 participants mostly already on statins. If approved, the daily pill could improve access to effective cholesterol treatment.
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Researchers from the University of Barcelona and the University of Oregon report that short DNA molecules known as polypurine reverse Hoogsteen hairpins (PPRHs) suppressed the PCSK9 gene and reduced blood cholesterol in a mouse model. In transgenic mice carrying the human PCSK9 gene, a single injection of one candidate (HpE12) cut plasma PCSK9 by 50% and total cholesterol by 47% three days later, according to findings published in Biochemical Pharmacology.
Researchers in Australia have found that a mysterious FDA-approved drug, called compound X, removes toxic alpha-synuclein proteins from the brains of mice with Parkinson's-like symptoms. The treatment improved the animals' balance and mobility by enhancing the brain's glymphatic waste disposal system. The findings were presented at a symposium in the UK.
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A large review of cardiovascular outcome trials found that people taking GLP-1 receptor agonists—drugs that include semaglutide (sold as Ozempic)—had a lower risk of major heart-related events than those given placebo. The analysis pooled results from 11 trials involving more than 90,000 participants, with an average follow-up of nearly three years, and reported benefits across patient subgroups including those with and without diabetes.