Safety and Tolerability of Intra-Articular Injection of Adipose-Derived Mesenchymal Stem Cells GXCPC1 in 11 Subjects With Knee Osteoarthritis: A Nonrandomized Pilot Study Without a Control Arm

• Published in: Cell transplantation Vol. 33; p. 9636897231221882
• Main Authors: Chen, Cheng-Fong, Chen, Yi-Chung, Fu, Yu-Show, Tsai, Shang-Wen, Wu, Po-Kuei, Chen, Chao-Ming, Chen, Wei-Ming, Wu, Hung-Ta Hondar, Lee, Chia-Hsin, Chang, Chao-Liang, Lin, Po-Cheng, Kao, Yong-Cheng, Chen, Chun-Hung, Chuang, Ming-Hsi
• Format: Journal Article
• Language: English
• Published: Los Angeles, CA SAGE Publications 01.01.2024; Sage Publications Ltd; SAGE Publishing
• Subjects: Adverse events; Humans; Injections, Intra-Articular; Knee; Mesenchymal Stem Cells; Osteoarthritis; Osteoarthritis, Knee - therapy; Pain; Pilot Projects; Stem cells; Visual perception; GXCPC1; knee osteoarthritis; allogeneic adipose-derived mesenchymal stem cells (ADSCs); safety; tolerability
• ISSN: 0963-6897; 1555-3892; 1555-3892
• DOI: 10.1177/09636897231221882

The current study aimed to determine the safety profile of intra-articular-injected allogeneic adipose-derived mesenchymal stem cells (ADSCs) GXCPC1 in subjects with knee osteoarthritis (OA) and its preliminary efficacy outcome. The 3 + 3 phase I study was designed with two dose-escalation cohorts: low dose (6.7 × 106 GXCPC1, N = 5) and high dose (4 × 107 GXCPC1, N = 6). The primary endpoint was safety, which was evaluated by recording adverse events throughout the trial; the secondary endpoints included total, pain, stiffness, and function subscales of the Western Ontario and McMaster Universities Arthritis Index (WOMAC), Visual Analogue Scale (VAS) for pain, and 12-Item Short Form (SF-12) health survey questionnaire. The GXCPC1 treatment was found to be safe after 1 year of follow-up with no treatment-related severe adverse events observed. When compared to baseline, subjects in both the low- and high-dose cohorts demonstrated improving trends in pain and knee function after receiving GXCPC1 treatment. Generally, the net change in pain (95% confidence interval (CI) = −7.773 to −2.561t at 12 weeks compared to baseline) and knee function (95% CI = −24.297 to −10.036t at 12 weeks compared to baseline) was better in subjects receiving high-dose GXCPC1. Although this study included a limited number of subjects without a placebo arm, it showed that the intra-articular injection of ADSCs was safe and well-tolerated in subjects with therapeutic alternatives to treat knee OA. However, a larger scale study with an appropriate control would be necessary for clinical efficacy in the following study.