Bad cholesterol slashed 62% by single dose of gene-editing drug in small trial
摘要
一项实验性基因编辑疗法VERVE-102在早期临床试验中取得积极进展。针对35名患者的I期安全性试验中期结果显示，该药物通过单次输注长期降低坏胆固醇，未出现严重不良事件，仅观察到一过性轻度肝酶升高。最高剂量组受试者的低密度脂蛋白（LDL）平均下降62%，降至78 mg/dL。若效果持续20年以上，可将动脉斑块相关心血管疾病风险降低约50%。目前最长随访18个
An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial.
Researchers running a Phase I safety trial for the drug, dubbed VERVE-102, published interim results from just 35 patients this week in the New England Journal of Medicine. Though the numbers are small and the analysis is preliminary, VERVE-102 appeared safe, with no serious adverse events reported from the treatment, even at the largest doses. The most significant finding was a temporary, mild increase of a liver enzyme that suggested minor injury in the liver, where the drug works.
The small amount of data also hints that the drug is effective. The subgroup of participants who received the largest dose have seen their bad cholesterol—that is, their low-density lipoprotein or LDL—drop 62 percent, to a mean of 78 mg per deciliter. For people with high cholesterol—like the participants in the trial—a reduction of this magnitude could cut the risk of cardiovascular disease from plaque buildup in arteries by an estimated 50 percent if it's sustained for over 20 years. The trial only has up to 18 months of follow-up data so far, but from that, the positive effects of VERVE-102 seem to be holding up. The LDL reductions have been sustained in all the subgroups.
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