百时美施贵宝披露细胞疗法早期试验一名患者死亡：输注44天后死于败血性肺炎
美国制药公司百时美施贵宝（Bristol Myers Squibb）10月23日在一场学术会议公布试验数据时披露，其实验性CAR-T细胞疗法在一项早期试验中有一名患者死亡。据路透社报道，死亡患者生前患有高度活跃的复发型多发性硬化症，接受该疗法（zolacabtagene autoleucel，简称zola-cel）输注44天后、并在接受大剂量化疗一天后，死于败血性肺炎。
这项早期试验共入组66名复发或进展型多发性硬化症患者。公司称，死亡患者此前已接受过大量治疗，输注后出现迟发的运动症状且对治疗无反应，又并发一系列其他问题；公司发言人表示：「患者出现了迟发的副作用和一系列其他并发症，且在接受CAR-T之前已接受过大量治疗，这使我们难以确定问题根源。」发言人同时表示，这例死亡与公司近期对zola-cel其他研究的暂停无关。百时美施贵宝今年9月主动暂停了zola-cel的相关研究以审查安全性数据，此前诺华（Novartis）已暂停其一款实验性细胞疗法的八项临床试验。
zola-cel靶向CD19蛋白，该蛋白存在于被认为在自身免疫性疾病中发挥作用的某些细胞上。公司表示，该试验中多数副作用短暂且可逆，与此前公布的zola-cel数据大体一致，结果仍支持该疗法「重置」自身免疫性疾病患者免疫系统的潜力。百时美施贵宝正与美国食品药品监管局（FDA）合作以重启全部研究，目标在2027年初恢复入组。消息公布后，公司股价周五下跌逾2%。
Bristol Myers Squibb Discloses Patient Death in Early-Stage Cell Therapy Trial: Septic Pneumonia 44 Days After Infusion
Bristol Myers Squibb disclosed on October 23, in a trial data readout at a conference, that a patient had died in an early-stage trial of its experimental CAR-T cell therapy. According to Reuters, the patient, who had highly active relapsing multiple sclerosis, died of septic pneumonia one day after receiving high-dose chemotherapy and 44 days after infusion of the therapy, zolacabtagene autoleucel, or zola-cel.
The early-stage trial enrolled 66 patients with relapsing or progressive forms of multiple sclerosis. The company said the patient who died had been heavily pretreated before receiving CAR-T, developed delayed motor symptoms that did not respond to treatment, and suffered a series of other complications. "The patient had a late onset side effect, a series of other complications and was heavily pretreated prior to CAR T, confounding our ability to determine the root cause of the issue," a company spokesperson said. The spokesperson said the death was unrelated to the company's recent voluntary pause on other zola-cel studies. Bristol Myers Squibb paused zola-cel studies in September to review safety data, after Novartis halted eight clinical trials of its own experimental cell therapy.
Zola-cel targets CD19, a protein found on certain cells believed to play a role in autoimmune diseases. The company said most side effects in the trial were brief and reversible, generally consistent with previously reported zola-cel data, and that the findings support the therapy's potential to reset the immune system in people with autoimmune diseases. Bristol Myers Squibb is working with the U.S. Food and Drug Administration (FDA) to restart all of the studies and aims to resume enrollment in early 2027. The drugmaker's shares were trading more than 2% lower on Friday.
来源：GSM·美国（原文链接）
图片来源：百时美施贵宝位于 New Jersey 州 Lawrenceville 的研发园区（资料图）。照片：Coolcaesar，CC BY-SA 3.0，经 Wikimedia Commons。 / Bristol Myers Squibb research campus in Lawrenceville, New Jersey (file photo). Photo: Coolcaesar, CC BY-SA 3.0, via Wikimedia Commons.
