Mesenchymal Stem Cells Infusion in Patients With Autoimmune Diseases

Summary

The goal of this study is to learn if mesenchymal stem cell therapy (treatment group) can effectively treat autoimmune diseases, when compared to normal saline (given to placebo group). The primary outcome measures will be clinical improvement based on the respective disease specific clinical scores, normalization of T-lymphocyte subsets and \> 50% reduction in disease specific antibody titres. The study will also document the type and frequency of any adverse event or side effects, reported by or seen in any of the trial participants. Patients in treatment group will receive single session of MSC therapy and placebo group will receive 0.9% saline solution. The participants will be followed at 3 and 6 months.

Timeline

Start

2025-02-01

Primary completion

2025-02-01

Completion

2026-02-01

Publications

• Background Le Blanc K, Frassoni F, Ball L, Locatelli F, Roelofs H, Lewis I, Lanino E, Sundberg B, Bernardo ME, Remberger M, Dini G, Egeler RM, Bacigalupo A, Fibbe W, Ringden O; Developmental Committee of the European Group for Blood and Marrow Transplantation. Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study. Lancet. 2008 May 10;371(9624):1579-86. doi: 10.1016/S0140-6736(08)60690-X.
• Background Laflamme MA, Murry CE. Regenerating the heart. Nat Biotechnol. 2005 Jul;23(7):845-56. doi: 10.1038/nbt1117.
• Background Viswanathan S, Shi Y, Galipeau J, Krampera M, Leblanc K, Martin I, Nolta J, Phinney DG, Sensebe L. Mesenchymal stem versus stromal cells: International Society for Cell & Gene Therapy (ISCT(R)) Mesenchymal Stromal Cell committee position statement on nomenclature. Cytotherapy. 2019 Oct;21(10):1019-1024. doi: 10.1016/j.jcyt.2019.08.002. Epub 2019 Sep 13.
• Background Choi YH, Kurtz A, Stamm C. Mesenchymal stem cells for cardiac cell therapy. Hum Gene Ther. 2011 Jan;22(1):3-17. doi: 10.1089/hum.2010.211.
• Background Nauta AJ, Fibbe WE. Immunomodulatory properties of mesenchymal stromal cells. Blood. 2007 Nov 15;110(10):3499-506. doi: 10.1182/blood-2007-02-069716. Epub 2007 Jul 30.
• Background Lee OK, Kuo TK, Chen WM, Lee KD, Hsieh SL, Chen TH. Isolation of multipotent mesenchymal stem cells from umbilical cord blood. Blood. 2004 Mar 1;103(5):1669-75. doi: 10.1182/blood-2003-05-1670. Epub 2003 Oct 23.
• Background Owen M, Friedenstein AJ. Stromal stem cells: marrow-derived osteogenic precursors. Ciba Found Symp. 1988;136:42-60. doi: 10.1002/9780470513637.ch4.
• Background Kabat M, Bobkov I, Kumar S, Grumet M. Trends in mesenchymal stem cell clinical trials 2004-2018: Is efficacy optimal in a narrow dose range? Stem Cells Transl Med. 2020 Jan;9(1):17-27. doi: 10.1002/sctm.19-0202. Epub 2019 Dec 5.
• Background Mizuno H. Adipose-derived stem cells for tissue repair and regeneration: ten years of research and a literature review. J Nippon Med Sch. 2009 Apr;76(2):56-66. doi: 10.1272/jnms.76.56.
• Background Song H, Song BW, Cha MJ, Choi IG, Hwang KC. Modification of mesenchymal stem cells for cardiac regeneration. Expert Opin Biol Ther. 2010 Mar;10(3):309-19. doi: 10.1517/14712590903455997.
• Background Phinney DG, Prockop DJ. Concise review: mesenchymal stem/multipotent stromal cells: the state of transdifferentiation and modes of tissue repair--current views. Stem Cells. 2007 Nov;25(11):2896-902. doi: 10.1634/stemcells.2007-0637. Epub 2007 Sep 27.
• Background Choi EW, Shin IS, Lee HW, Park SY, Park JH, Nam MH, Kim JS, Woo SK, Yoon EJ, Kang SK, Ra JC, Youn HY, Hong SH. Transplantation of CTLA4Ig gene-transduced adipose tissue-derived mesenchymal stem cells reduces inflammatory immune response and improves Th1/Th2 balance in experimental autoimmune thyroiditis. J Gene Med. 2011 Jan;13(1):3-16. doi: 10.1002/jgm.1531.
• Background Cai L, Johnstone BH, Cook TG, Tan J, Fishbein MC, Chen PS, March KL. IFATS collection: Human adipose tissue-derived stem cells induce angiogenesis and nerve sprouting following myocardial infarction, in conjunction with potent preservation of cardiac function. Stem Cells. 2009 Jan;27(1):230-7. doi: 10.1634/stemcells.2008-0273.
• Background Augello A, Tasso R, Negrini SM, Cancedda R, Pennesi G. Cell therapy using allogeneic bone marrow mesenchymal stem cells prevents tissue damage in collagen-induced arthritis. Arthritis Rheum. 2007 Apr;56(4):1175-86. doi: 10.1002/art.22511.
• Background Zappia E, Casazza S, Pedemonte E, Benvenuto F, Bonanni I, Gerdoni E, Giunti D, Ceravolo A, Cazzanti F, Frassoni F, Mancardi G, Uccelli A. Mesenchymal stem cells ameliorate experimental autoimmune encephalomyelitis inducing T-cell anergy. Blood. 2005 Sep 1;106(5):1755-61. doi: 10.1182/blood-2005-04-1496. Epub 2005 May 19.
• Background Yanez R, Lamana ML, Garcia-Castro J, Colmenero I, Ramirez M, Bueren JA. Adipose tissue-derived mesenchymal stem cells have in vivo immunosuppressive properties applicable for the control of the graft-versus-host disease. Stem Cells. 2006 Nov;24(11):2582-91. doi: 10.1634/stemcells.2006-0228. Epub 2006 Jul 27.
• Background Garcia-Gomez I, Elvira G, Zapata AG, Lamana ML, Ramirez M, Castro JG, Arranz MG, Vicente A, Bueren J, Garcia-Olmo D. Mesenchymal stem cells: biological properties and clinical applications. Expert Opin Biol Ther. 2010 Oct;10(10):1453-68. doi: 10.1517/14712598.2010.519333.
• Background Parekkadan B, Milwid JM. Mesenchymal stem cells as therapeutics. Annu Rev Biomed Eng. 2010 Aug 15;12:87-117. doi: 10.1146/annurev-bioeng-070909-105309.
• Background Si YL, Zhao YL, Hao HJ, Fu XB, Han WD. MSCs: Biological characteristics, clinical applications and their outstanding concerns. Ageing Res Rev. 2011 Jan;10(1):93-103. doi: 10.1016/j.arr.2010.08.005. Epub 2010 Aug 19.
• Background Mohsin Z, Asghar AA, Faiq A, Khalid I, Ul-Haque I, Rehman S, Ahmed SI, Basalat ST, Aimen A, Shafique S, Hanif A, Iqbal MW, Samad SA, Siddiqui F, Hameed I, Safri M. Prevalence of Rheumatic Diseases in a Tertiary Care Hospital of Karachi. Cureus. 2018 Jun 22;10(6):e2858. doi: 10.7759/cureus.2858.
• Background Cooper GS, Stroehla BC. The epidemiology of autoimmune diseases. Autoimmun Rev. 2003 May;2(3):119-25. doi: 10.1016/s1568-9972(03)00006-5.

Drugs

Indications