Nadofaragene firadenovec:膀胱肿瘤领域的突破

Abdullah Nadeem, Khulud Qamar, Wajeeha Bilal, Laiba Imran Vohra, Areeba Ahsan, Rabeea Tariq
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引用次数: 0

摘要

肌肉侵袭性膀胱肿瘤由于其倾向于远处转移而造成严重的死亡风险。这类肿瘤的治疗范围包括手术、化疗和放疗,根据癌症的严重程度量身定制。在高风险卡介苗(BCG)无反应的非肌肉浸润性膀胱癌(NMIBC)的背景下,一种新的治疗方法作为希望的灯塔出现了。Nadofaragene firadenovec是一种开创性的基因疗法,已获得全球批准用于治疗膀胱癌,标志着膀胱癌治疗的分水岭时刻。Nadofaragene firadenovec被巧妙地设计用于治疗高危bcg无反应的NMIBC,特别是成年患者的原位癌(CIS)伴或不伴乳头状肿瘤。该疗法以载体DNA为基础,编码干扰素(IFN)-2b,使尿路上皮细胞具有产生IFN-2b的能力。由此产生的一系列事件触发了对癌症的多方面攻击,其特点是具有免疫刺激、抗血管生成和细胞凋亡的作用。nadofaragene firadenovec的治疗效果取决于其利用转化的尿路上皮细胞递送这些靶向抗癌活性的能力。nadofaragene firadenovec的进化轨迹在2022年12月获得美国批准时达到了顶峰,标志着该领域的关键时刻。值得注意的是,部分患者(约30%)对卡介苗治疗难治性。对于这些人来说,替代治疗途径是必要的。目前,对于非肌性浸润性膀胱癌患者,缺乏一个明确的、持久的解决方案。在此背景下,nadofaragene firadenovec的引入预示着一种经批准的治疗干预措施在全球范围内的可用性迈出了重大的一步。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Nadofaragene firadenovec: a breakthrough in the field of bladder oncology
Muscle-invasive bladder tumors pose a grave mortality risk due to their propensity for distant metastases. The therapeutic spectrum for such tumors encompasses surgery, chemotherapy, and radiation, tailored to the cancer’s severity. In the context of high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC), a novel treatment has emerged as a beacon of hope. Nadofaragene firadenovec, a pioneering gene therapy, has gained worldwide approval for combating this condition, marking a watershed moment in bladder cancer therapy. Nadofaragene firadenovec is ingeniously designed to address high-risk BCG-unresponsive NMIBC, particularly carcinoma in situ (CIS) with or without papillary tumors, in adult patients. Rooted in a vector DNA, this therapy encodes interferon (IFN)-2b, which imparts urothelial cells with the ability to generate IFN-2b. The resulting cascade of events triggers a multifaceted assault on cancer, characterized by its immunostimulatory, antiangiogenic, and apoptotic effects. The therapeutic efficacy of nadofaragene firadenovec rests on its capacity to exploit the transformed urothelial cells to deliver these targeted anticancer activities. The evolutionary trajectory of nadofaragene firadenovec culminated in its monumental approval in December 2022 by the United States, signifying a pivotal juncture in the field. Notably, a segment of patients, approximately 30%, prove refractory to BCG treatment. For these individuals, alternative therapeutic avenues are imperative. Presently, the landscape for patients with non-muscle invasive bladder cancer lacks a definitive, enduring solution. Against this backdrop, the introduction of nadofaragene firadenovec heralds a momentous stride toward the global availability of an authorized therapeutic intervention.
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