ACR Meeting Abstracts

ACR Meeting Abstracts

Abstract Number: 2623

Intravenous Infusion of Umbilical Cord Blood-Derived Mesenchymal Stem Cells in Rheumatoid Arthritis: A Phase 1, Proof-of-Concept Clinical Trial

Dong Jin Go1, Hee-suk Lim2, Ahrmi Cho3, Kyounghwan Roh3, Kwang-Won Seo3, Kyung-Sun Kang3,4 and Kichul Shin5, 1Department of Molecular Medicine and Biopharmaceutical Sciences, Graduate School of Convergence Science and Technology, Seoul National University, Seoul, Korea, The Republic of, 2Division of Rheumatology, Department of Internal Medicine, Seoul Metropolitan Government-Seoul National University Boramae Medical Center, Seoul, South Korea, 3Institute for Stem cell Regenerative Medicine, Kangstem Biotech, Seoul, Korea, The Republic of, 4Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul, Korea, The Republic of, 5Kyungnam villa #102, Division of Rheumatology, Department of Internal Medicine, SMG-SNU Boramae Medical Center, Seoul, Korea, Republic of

Meeting: 2016 ACR/ARHP Annual Meeting

Date of first publication: September 28, 2016

Keywords: , ,

Session Information

Date: Tuesday, November 15, 2016

Title: Rheumatoid Arthritis – Small Molecules, Biologics and Gene Therapy - Poster III

Session Type: ACR Poster Session C

Session Time: 9:00AM-11:00AM

Background/Purpose: The immunomodulatory actions of human umbilical cord blood (hUCB)-derived mesenchymal stem cells (MSCs) in rheumatoid arthritis (RA) have been studied extensively via in vitro or pre-clinical studies, yet few human trials have been conducted investigating the outcome of hUCB-derived MSC infusion.

Methods: The CURE-iv (Clinical and safety assessment of human Umbilical cord blood-derived mesenchymal stem cell therapy for RhEumatoid arthritis patients administered intravenously) trial was a phase 1, proof-of-concept clinical trial for RA patient with moderate disease activity despite treatment with methotrexate. Patients meeting the 2010 ACR/EULAR classification criteria and with a DAS28-ESR >3.2 were eligible for the trial. Subjects were each given a single intravenous infusion of 2.5 x 107, or 5 x 107, or 1 x 108 cells of hUCB-derived MSCs for 30 minutes; 3 patients in each cluster, with increment of cell numbers when there was no dose-limited adverse events. Clinical and safety parameters were monitored and followed after the infusion period (first 24 hours, 72 hours, 1 week, and 4 weeks). Serum cytokines at baseline and 24 hours after the infusion were analyzed.

Results: Eleven RA patients were screened, 9 of which were enrolled from a single center. The mean age was 57.4 years, 78 % being female, with a disease duration of 9.5 ± 8.7 years, and DAS28-ESR 4.53 ± 1.35. There was no ominous safety signal in all clusters up to 4 weeks after the infusion. One patient stated joint pain 60 min after the infusion (5 x 107 group), but it was thought to be unrelated to the investigational product. ESR and CRP changes at 4 weeks (n=9) were -7.89 ± 10.36 (p= 0.0517), and -0.37 ± 1.09 (p=0.3362). DAS28 and HAQ changes at 4 weeks were -1.60 ± 1.57 (p= 0.0159), and -0.15 ± 0.48 (p= 0.3706), respectively. One patient in the 1 x 108 group showed substantial decrease in all serum levels of IL-1b, IL-6, IL-8, and TNF-a.

Conclusion: This phase 1 clinical trial – a single dose intravenous infusion of hUCB-derived MSCs – for established RA patients was completed without any short-term safety concerns (NCT02221258).

Disclosure: D. J. Go, None; H. S. Lim, None; A. Cho, Kangstem Biotech, 3; K. Roh, Kangstem Biotech, 3; K. W. Seo, Kangstem Biotech, 3; K. S. Kang, Kangstem Biotech, 4; K. Shin, None.

To cite this abstract in AMA style:

Go DJ, Lim HS, Cho A, Roh K, Seo KW, Kang KS, Shin K. Intravenous Infusion of Umbilical Cord Blood-Derived Mesenchymal Stem Cells in Rheumatoid Arthritis: A Phase 1, Proof-of-Concept Clinical Trial [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/intravenous-infusion-of-umbilical-cord-blood-derived-mesenchymal-stem-cells-in-rheumatoid-arthritis-a-phase-1-proof-of-concept-clinical-trial/. Accessed .

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