A single-center, phase 1/2a trial of hESC-derived mesenchymal stem cells (MR-MC-01) for safety and efficacy in interstitial cystitis patients.

IF 5.4 2区 医学 Q1 CELL & TISSUE ENGINEERING
Yoon Soo Kyung, Ki-Sung Hong, Hyung-Min Chung, Jung Hyun Shin, Myung-Soo Choo, Eun-Young Kim, Jeong Min Shin, Ah Reum Kang, Jin Won Seo, Juhyun Park, Se-Pill Park
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Abstract

This study investigated the safety and efficacy of MR-MC-01, a mesenchymal stem cell therapy derived from human embryonic stem cells, in patients with interstitial cystitis (IC), particularly those with Hunner lesions unresponsive to pentosan polysulfate sodium (PPS). Conducted as a prospective, randomized, double-blind, placebo-controlled phase I/IIa clinical trial, it enrolled 22 patients, with six completing phase I and 16 participating in phase IIa. Phase I tested 2 doses (2.0 × 107 and 5.0 × 107 cells) to determine the maximum tolerated dose (MTD), revealing no dose-limiting toxicities and only mild adverse events such as transient hemorrhage and bladder pain. In phase IIa, 12 participants received the MTD of 5.0 × 107 cells, and 4 received placebo. Significant reductions in interstitial cystitis questionnaire (ICQ) and pain urgency frequency (PUF) scores were observed in the treatment group. Improvements were noted in nocturnal voiding frequency and Hunner lesion size, with 8 patients showing either a reduction or complete resolution of lesions after 6 months. The global response assessment (GRA) reported moderate to marked improvement in 41.67% of treated patients versus 25% in the placebo group. MR-MC-01 demonstrated no serious drug-related adverse events, highlighting its favorable safety profile. These findings suggest that MR-MC-01 not only alleviates symptoms but also promotes structural recovery in IC, making it a promising treatment option. Further large-scale, long-term studies are warranted to confirm these results and optimize therapeutic protocols. (Identifier: NCT04610359).

hesc来源的间充质干细胞(MR-MC-01)在间质性膀胱炎患者中的安全性和有效性的单中心,1/2a期试验。
本研究探讨了源自人胚胎干细胞的间充质干细胞疗法MR-MC-01治疗间质性膀胱炎(IC)患者的安全性和有效性,特别是那些对戊聚糖聚硫酸钠(PPS)无反应的Hunner病变患者。作为一项前瞻性、随机、双盲、安慰剂对照的I/IIa期临床试验,该研究纳入了22例患者,其中6例完成I期,16例参与IIa期。I期试验了2个剂量(2.0 × 107和5.0 × 107细胞)来确定最大耐受剂量(MTD),结果显示没有剂量限制性毒性,只有轻微的不良事件,如短暂性出血和膀胱疼痛。在IIa期,12名参与者接受MTD为5.0 × 107个细胞,4名接受安慰剂。治疗组间质性膀胱炎问卷(ICQ)和疼痛急迫频率(PUF)评分显著降低。夜间排尿频率和Hunner病变大小均有改善,其中8例患者在6个月后病变减轻或完全消退。总体反应评估(GRA)报告41.67%的治疗患者有中度至显著改善,而安慰剂组为25%。MR-MC-01未出现严重的药物相关不良事件,突出了其良好的安全性。这些发现表明MR-MC-01不仅可以缓解症状,还可以促进IC的结构恢复,使其成为一种有希望的治疗选择。需要进一步的大规模长期研究来证实这些结果并优化治疗方案。(标识符:NCT04610359)。
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来源期刊
Stem Cells Translational Medicine
Stem Cells Translational Medicine CELL & TISSUE ENGINEERING-
CiteScore
12.90
自引率
3.30%
发文量
140
审稿时长
6-12 weeks
期刊介绍: STEM CELLS Translational Medicine is a monthly, peer-reviewed, largely online, open access journal. STEM CELLS Translational Medicine works to advance the utilization of cells for clinical therapy. By bridging stem cell molecular and biological research and helping speed translations of emerging lab discoveries into clinical trials, STEM CELLS Translational Medicine will help move applications of these critical investigations closer to accepted best patient practices and ultimately improve outcomes. The journal encourages original research articles and concise reviews describing laboratory investigations of stem cells, including their characterization and manipulation, and the translation of their clinical aspects of from the bench to patient care. STEM CELLS Translational Medicine covers all aspects of translational cell studies, including bench research, first-in-human case studies, and relevant clinical trials.
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