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New Discoveries-Clinical Outcomes DMD Stem Cell Therapy Hilal Kalkan(1),et al. , Targeting gut dysbiosis against inflammation and impaired autophagy in Duchenne muscular dystrophy EMBO Molecular Medicine (2023) Olivier Boyer (1) et al, Myogenic Cell Transplantation in Genetic and Aquired Diseases of Skeletal Muscles Frontiers in Genetics (2023)
Table X. Safety and efficacy outcomes of DMD clinical investigations with stem cells.
(b) Sharma et al., A clinical study shows safety and efficacy of autologous bone marrow mononuclear cell therapy to improve quality of life in muscular dystrophy patients. Cell Transplant. 2013;22 Suppl 1:S127-38. doi: 10.3727/096368913X672136. Epub 2013 Sep 10.PMID: 24070109. (c) Sharma et al., Autologous bone marrow mononuclear cell transplantation in Duchenne muscular dystrophy - a case report. Am J Case Rep. 2014 Mar 28;15:128- 34. doi: 10.12659/AJCR.890078. eCollection 2014.PMID: 24711886. (d) Sych et al., Efficacy of Fetal Stem Cells in Duchenne Muscular Dystrophy Therapy. Journal of Neurorestoratology 2014:2 37-46 (e) Dai et al., Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy - Phase I-II. Degener Neurol Neuromuscul Dis. 2018 Oct 26;8:63-77. doi: 10.2147/DNND.S170087. eCollection 2018.PMID: 30498389. (f) Klimczak et al., Co-transplantation of Bone Marrow-MSCs and Myogenic stem/progenitor cells from adult donors improves muscle function of patients with Duchenne Muscular Dystrophy. Cells, 2020 Apr 30;9(5):1119. doi: 10.3390/cells9051119. INDIA A Clinical Study Shows Safety and Efficacy of Autologous Bone Marrow Mononuclear Cell Therapy to Improve Quality of Life in Muscular Dystrophy Patients. Cell Transplantation, Vol.22, Supplement 1, pp. S127-S138, 2013 0963-6897/13 $90.00 + .00 Printed in the USA. All rights reserved. DOI: https://journals.sagepub.com/doi/10.3727/096368913X672136 Copyright 2013 Cogizant Comm. Corp. E_ISSN 1555-3892 Sharma A. et al,: Cellular therapy for Duchenne Muscular Dystrophy. (c) AM J Case Rep, 2014; 15: 128-134 Outcome: Indian doctors continue to apply stem cell therapies in the clinic for Duchenne mitigation. Autologous stem cell administration for Duchenne muscular dystrophy is now the standard of care in India. The Neurogen Brain and Spine Clinic has treated over 1000 Duchenne patients with bone marrow derived stem cells and, in peer-reviewed journal research, has estimated a life extension of more than five years. No country has a similar record or data. POLAND (2020) Klimczak, A., Zimna, A., Malcher, A., Kozlowska, U., Futoma, K., and Czarnota, J. (2020). Co-transplantation of bone marrow-MSCs and Myogenic stem/progenitor cells from adult donors improves muscle function of patients with Duchenne Muscular Dystrophy. cells 9:1119. doi: 10.3390/cells9051119 (2021) Swiatkowska-Flis B, Zdolinska-Malinowska I, Slugocka D, Boruczkowski D. The use of umbilical cord-derived mesenchymal stem cells in patients with muscular dystrophies: Results from compassionate use in real-life settings. STEM CELLS Transl Med. 2021;1-12. https://academic.oup.com/stcltm/article/10/10/1372/6515807 Outcome: In this study, we administered advanced therapy medicinal product containing umbilical cord-derived mesenchymal stem cells (UC_MSCs) to 22 patients with muscular dystrophies. Patients received one to five intravenous and/or intrathecal injections per treatment course in up to two courses every two months. Four standard doses of 10, 20, 30, or 40 106 UC-MSCs per injection were used; the aproximate dose per kilogram was 1 106 UC- MSCs. Muscle strength was measured with a set of CQ Dynamometer computerized force meters (CQ Elektronik System, Czernica, Poland). Statistical analysis of muscle strength in the whole group showed significant improvement in the right upper limb (+4.0 N); left hip straightening (+4.5 N) and adduction (+0.5 N); right hip straightening (+1.0 N), bending (+7.5 N), and adduction (+2.5 N); right knee straightening (+8.5 N); left shoulder revocation (=13.0 N), straightening (+5.5 N), and bending (+6.5 N); right shoulder adduction (+3.0 N), revocation (+10.5 N), and bending (+5 N); and right elbow straightening (+9.5 N); all these differences were statistically significant. TURKEY Dai, A., Baspinar, O., Yesilyurt, A., Sun, E., Inci Ayedemir, C., and Otzel, O. N. (2018). Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne Muscular Dystrophy; Phase I/II. Dai, A., Baspinar, O., Yesilyurt, A., Sun, E., Inci Aydemir, C., and Otzel, O. N. (2018). Outcome: (Nine patients total, these are representative) Patient 1 Based on measurements taken after the first injection, the patient's FVC had increased up to 23% in the second month and raised to 57% at the end of the ninth month. The FEV1 level had increased up to 9% in the second month after the ninth month, it raised to 41%. Patient 2 After the first injection, patient number 2's FVC had increased up to 12% in the second month and raised to 28% at the end of the ninth month. Accordingly, the FEV1 level had increased up to 4% and 32% in the same period of FVC. The EF level had dropped down to 10% at the end of the second month and 3% at the end of the ninth month. Patient 4 In respect to the respiratory tests, the patient's FVC had increased up to 7% in the second month and raised to 57% at the end of the ninth month after the first MSC injection. The FEV1 level had increased up to 6% in the second month and after the ninth month it raised to 58%. The EF level had dropped down to 14% at the end of thesecond month and 15% at the end of the ninth month. Although an increaase of 6% was measured for CK level at the end of the first year after the beginning of the treatment, a dramatic decrease of 66% was measured at the initiation of the treatment, a dramatic reduction of 97% at the end of the first year and 62% at the end of the second year were measured by means of CK levels. UNITED STATES Mahir Mohiuddin 2,4,† , Jeongmoon J. Choi 1,4,† , Nan Hee Lee 1,4 , Hyeonsoo Jeong 1,4 , Shannon E. Anderson 2,4 , Woojin M. Han 3,4 , Berna Aliya 1,4 Tsvetomira Z. Peykova 1,2 , Sumit Verma 6 , Andrés J. García 3,4 , Carlos A. Aguilar 5 , and Young C. Jang 1,2,4,* Transplantation of Muscle Stem Cell Mitrochondria Rejuvenates the Bioenergetic Function of Dystrophic Muscle (2020) bioRxiv preprint doi: https://www.biorxiv.org/content/10.1101/2020.04.17.017822v1; this version posted April 18, 2020. The copyright holder for this preprint (which was not certified by peer review) is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. It is made available under a CC-BY_NC_ND 4.0 International license Tripodi, L., Villa, C., Molinaro, D., Torrente, Y., Farini, A., The Immune System in Duchenne Muscular Dystrophy Pathogenesis. Biomedicines 2021, 9, 1447. https://doi.org/10.3390/biomedicines9101447 KOREA Choi, A., Park, S. E., Bin Jeong, j., Choi, S. J., Oh, S. Y., and Ryu, G., H., (2020) Antifibrotic effect of human Wharton's Jelly-derived mesenchymal stem cells on skeletal muscle cells, mediated by secretion of MMP-1. Int. J. Mol. Sci. 21:6269. https://www.mdpi.com/1422-0067/21/17/6269
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