Berry JD, Cudkowicz ME, Windebank AJ, Staff NP, Owegi M, Nicholson K, McKenna-Yasek D, Levy YS, Abramov N, Kaspi H, Mehra M, Aricha R, Gothelf Y, Brown RH. NurOwn, phase 2, randomized, clinical trial in patients with ALS: Safety, clinical, and biomarker results. Neurology. 2019 Dec 10;93(24):e2294-e2305. doi: 10.1212/WNL.0000000000008620. Epub 2019 Nov 18. PmiD: 31740545; PMCID: PMC6937497.
Cudkowicz ME, Lindborg SR, Goyal NA, miller RG, Burford MJ, Berry JD, Nicholson KA, Mozaffar T, Katz JS, Jenkins LJ, Baloh RH, Lewis RA, Staff NP, Owegi MA, Berry DA, Gothelf Y, Levy YS, Aricha R, Kern RZ, Windebank AJ, Brown RH Jr. A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis. Muscle Nerve. 2022 Mar;65(3):291-302. doi: 10.1002/mus.27472. Epub 2022 Jan 5. PmiD: 34890069; PMCID: PMC9305113.
Mazzini L, Ferrari D, Andjus PR, et al. Advances in stem cell therapy for amyotrophic lateral sclerosis. Expert Opin Biol Ther. 2018;18(8):865-881. doi:10.1080/14712598.2018.1503248
Moura, M. C., Novaes, M. R. C. G., Zago, Y. S., Eduardo, E. J., & Casulari, L. A. (2016). Efficacy of stem cell therapy in amyotrophic lateral sclerosis: a systematic review and meta-analysis. Journal of clinical medicine research, 8(4), 317.
Abdul Wahid SF, Law ZK, Ismail NA, Azman Ali R, Lai NM. Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev. 2016;11:CD011742. doi:10.1002/14651858.CD011742.pub2
Aljabri A, Halawani A, Bin Lajdam G, Labban S, Alshehri S, Felemban R. The Safety and Efficacy of Stem Cell Therapy as an Emerging Therapy for ALS: A Systematic Review of Controlled Clinical Trials. Front Neurol. 2021 Dec 1;12:783122. doi: 10.3389/fneur.2021.783122. PmiD: 34938264; PMCID: PMC8685950.
Barczewska M, Maksymowicz S, Zdolińska-Malinowska I, Siwek T, Grudniak M. Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study. Stem Cell Rev Rep. 2020 Oct;16(5):922-932. doi: 10.1007/s12015-020-10016-7. PmiD: 32725316; PMCID: PMC7456414.
Feldman EL, Boulis NM, Hur J, Johe K, Rutkove SB, Federici T, Polak M, Bordeau J, Sakowski SA, Glass JD. Intraspinal neural stem cell transplantation in amyotrophic lateral sclerosis: Phase 1 trial outcomes. Annals of Neurology. 2014 Mar;75(3):363-73. doi: 10.1002/ana.24113
Morata-Tarifa C, Azkona G, Glass J, Mazzini L, Sanchez-Pernaute R. Looking backward to move forward: a meta-analysis of stem cell therapy in amyotrophic lateral sclerosis. npj Regenerative Medicine. 2021 Apr 1;6(1).
Chen L, Huang H, Zhang J, Zhang F, Liu Y, Xi H, Wang H, Gu Z, Song Y, Li Y, Tan K. Short-term outcome of olfactory ensheathing cells transplantation for treatment of amyotrophic lateral sclerosis. Zhongguo Xiu Fu Chong Jian Wai Ke Za Zhi. 2007 Sep;21(9):961-6. PubMed PmiD: 17933231.
Mazzini L, Fagioli F, Boccaletti R, Mareschi K, Oliveri G, Olivieri C, Pastore I, Marasso R, Madon E. Stem cell therapy in amyotrophic lateral sclerosis: a methodological approach in humans. Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders. 2003;4(3):158-161. doi: 10.1080/14660820310014653.
Martinez HR, Gonzalez-Garza MT, Moreno-Cuevas JE, Caro E, Gutierrez-Jimenez E, Segura JJ. Stem-cell transplantation into the frontal motor cortex in amyotrophic lateral sclerosis patients. Cytotherapy. 2009;11(1):26-34. doi:10.1080/14653240802644651
Deda H, Inci MC, Kürekçi AE, Sav A, Kayihan K, Ozgün E, et al. Treatment of amyotrophic lateral sclerosis patients by autologous bone marrow-derived hematopoietic stem cell transplantation: a 1-year follow-up. Cytotherapy. 2009;11(1):18–25.
Gamez J, Carmona F, Raguer N, Ferrer-Sancho J, Martín-Henao GA, Martí-Beltrán S, et al. Cellular transplants in amyotrophic lateral sclerosis patients: an observational study. Cytotherapy. 2010 Sep;12(5):669–77.
Karussis D, Karageorgiou C, Vaknin-Dembinsky A, Gowda-Kurkalli B, Gomori JM, Kassis I, et al. Safety and Immunological Effects of Mesenchymal Stem Cell Transplantation in Patients With Multiple Sclerosis and Amyotrophic Lateral Sclerosis. Arch Neurol. 2010 Oct;67(10):1187–94.
Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, et al. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar;73(3):337–44.
Riley J, Glass J, Feldman EL, Polak M, Bordeau J, Federici T, et al. Intraspinal stem cell transplantation in amyotrophic lateral sclerosis: a phase I trial, cervical microinjection, and final surgical safety outcomes. Neurosurgery. 2014 Jan;74(1):77–87.
Mazzini L, Mareschi K, Ferrero I, miglioretti M, Stecco A, Servo S, et al. Mesenchymal stromal cell transplantation in amyotrophic lateral sclerosis: a long-term safety study. Cytotherapy. 2012 Jan;14(1):56–60.
Blanquer M, Moraleda JM, Iniesta F, Gómez-Espuch J, Meca-Lallana J, Villaverde R, et al. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277–85.
Oh K-W, Moon C, Kim HY, Oh S-I, Park J, Lee JH, et al. Phase I trial of repeated intrathecal autologous bone marrow-derived mesenchymal stromal cells in amyotrophic lateral sclerosis. Stem Cells Transl Med. 2015 Jun;4(6):590–7.
Huang H, Chen L, Xi H, Wang H, Zhang J, Zhang F, et al. Fetal olfactory ensheathing cells transplantation in amyotrophic lateral sclerosis patients: a controlled pilot study. Clin Transplant. 2008 Dec;22(6):710–8.
Glass JD, Boulis NM, Johe K, Rutkove SB, Federici T, Polak M, et al. Lumbar intraspinal injection of neural stem cells in patients with amyotrophic lateral sclerosis: results of a phase I trial in 12 patients. Stem Cells. 2012 Jun;30(6):1144–51.
Rushkevich YN, Kosmacheva SM, Zabrodets GV, Ignatenko SI, Goncharova NV, Severin IN, et al. The Use of Autologous Mesenchymal Stem Cells for Cell Therapy of Patients with Amyotrophic Lateral Sclerosis in Belarus. Bull Exp Biol Med. 2015 Aug;159(4):576–81.
Ruiz-López FJ, Blanquer M. Autologous bone marrow mononuclear cells as neuroprotective treatment of amyotrophic lateral sclerosis. Neural Regen Res. 2016 Apr;11(4):568–9.
Syková E, Rychmach P, Drahorádová I, Konrádová Š, Růžičková K, Voříšek I, et al. Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial. Cell Transplant. 2017 Apr 13;26(4):647–58.
Feldman EL, Boulis NM, Hur J, Johe K, Rutkove SB, Federici T, Polak M, Bordeau J, Sakowski SA, Glass JD. Intraspinal neural stem cell transplantation in amyotrophic lateral sclerosis: phase 1 trial outcomes. Ann Neurol. 2014 Mar;75(3):363-73. doi: 10.1002/ana.24113. Epub 2014 Mar 7. PubMed PmiD: 24510776; PubMed Central PMCID: PMC4005820.
Chen L, Chen D, Xi H, Wang Q, Liu Y, Zhang F, Wang H, Ren Y, Xiao J, Wang Y, Huang H. Olfactory ensheathing cell neurorestorotherapy for amyotrophic lateral sclerosis patients: benefits from multiple transplantations. Cell Transplant. 2012;21 Suppl 1:S65-77. doi: 10.3727/096368912X633789. PubMed PmiD: 22507682.
Mazzini L, Gelati M, Profico DC, Sorarù G, Ferrari D, Copetti M, Muzi G, Ricciolini C, Carletti S, Giorgi C, Spera C, Frondizi D, Masiero S, Stecco A, Cisari C, Bersano E, De Marchi F, Sarnelli MF, Querin G, Cantello R, Petruzzelli F, Maglione A, Zalfa C, Binda E, Visioli A, Trombetta D, Torres B, Bernardini L, Gaiani A, Massara M, Paolucci S, Boulis NM, Vescovi AL; ALS-NSCs Trial Study Group. Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long-Term Outcome. Stem Cells Transl Med. 2019 Sep;8(9):887-897.
Karussis D, Karageorgiou C, Vaknin-Dembinsky A, Gowda-Kurkalli B, Gomori JM, Kassis I, et al. Safety and Immunological Effects of Mesenchymal Stem Cell Transplantation in Patients With Multiple Sclerosis and Amyotrophic Lateral Sclerosis. Arch Neurol. 2010 Oct;67(10):1187–94.
Petrou P, Kassis I, Yaghmour NE, Ginzberg A, Karussis D. A phase II clinical trial with repeated intrathecal injections of autologous mesenchymal stem cells in patients with amyotrophic lateral sclerosis. Front Biosci (Landmark Ed). 2021 Oct 30;26(10):693-706. doi: 10.52586/4980. PmiD: 34719198.
Siwek T, Jezierska-Woźniak K, Maksymowicz S, Barczewska M, Sowa M, Badowska W, Maksymowicz W. Repeat Administration of Bone Marrow-Derived Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis. Med Sci Monit. 2020 Dec 10;26:e927484. doi: 10.12659/MSM.927484. PmiD: 33301428; PMCID: PMC7737405.
Tavakol-Afshari J, Boroumand AR, Farkhad NK, Moghadam AA, Sahab-Negah S, Gorji A. Safety and efficacy of bone marrow derived-mesenchymal stem cells transplantation in patients with amyotrophic lateral sclerosis. Regenerative Therapy. 2021 Dec 1;18:268-74.
Martínez HR, Molina-Lopez JF, González-Garza MT, et al. Stem Cell Transplantation in Amyotrophic Lateral Sclerosis Patients: Methodological Approach, Safety, and Feasibility. Cell Transplantation. 2012;21(9):1899-1907. doi:10.3727/096368911X582769.
Appel SH, Engelhardt JI, Henkel JS, Siklos L, Beers DR, Yen AA, Simpson EP, Luo Y, Carrum G, Heslop HE, Brenner MK, Popat U. Hematopoietic stem cell transplantation in patients with sporadic amyotrophic lateral sclerosis. Neurology. 2008 Oct 20;71(17):1326-34.
Shigematsu K, Takeda T, Komori N, Urushihata N, Oki K, Tahara K, Yamagishi H. Long-term survival of a patient with amyotrophic lateral sclerosis (ALS) who received autologous adipose-derived mesenchymal stem cells. Eur Rev Med Pharmacol Sci. 2021 Jun 1;25(11):4086-90.
Ala'a, A. H., Elkins, J., & Hassan, H. Y. Case Report: Stem cell therapy in amyotrophic lateral sclerosis. F1000Research. 2022;10:1080.
Minguell, J. J., Allers, C., Jones, J. A., & Ganji, S. S. Allogeneic mesenchymal stem cells infusion to an ALS patient proved to be safe and capable to initiate clinical recuperation. Regen Med. 2013;9(5):627-647.
Brennan S, Keon M, Su Z, Liu B, Song XM, Zaw T, McKay M, Pascovici D, Saksena NK. Molecular Basis of Allogeneic Pluripotent Stem Cell Treatment of a Patient with Sporadic ALS. OBM Geriatrics. 2021;5(1):164. doi:10.21926/obm.geriatr.2101164.
Uriel Ayllón Álvarez D, Neri Calixto M, Humberto Aparicio Albarrán C, Mendieta Zerón H. Autologous Stem Cell Transplantation and Immunomodulation in Amyotrophic Lateral Sclerosis. MNJ (Malang Neurology Journal). 2021 Jul 1;7(2):140-5.
Mazzini, L., De Marchi, F., Buzanska, L., Follenzi, A., Glover, J. C., Gelati, M., ... & Ferrari, D. Current status and new avenues of stem cell-based preclinical and therapeutic approaches in amyotrophic lateral sclerosis. Expert Opin Biol Ther. 2024;24(9):933-954.
Nam, J. Y., Chun, S., Lee, T. Y., Seo, Y., Kim, K., Park, J., ... & Kim, S. H. Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: propensity-score-matched control, surveillance study. Front Aging Neurosci. 2023;15:1148444.
Şenkal-Turhan, S., Bulut-Okumuş, E., Şahin, F., Yavuz, Y., Yılmaz, B., Şişli, H. B., ... & Doğan, A. Derivation of functional neurons from induced pluripotent stem cells using a simple neuromesodermal progenitor generation and rapid spinal cord neuron differentiation process. Human Cell. 2025;38(3):69.