Results from phase I clinical trial with intraspinal injection of neural stem cells in amyotrophic lateral sclerosis: a long-term outcome
Open accessAbstract
The main objective of this phase I trial was to assess the feasibility and safety of microtransplanting human neural stem cell (hNSC) lines into the spinal cord of patients with amyotrophic lateral sclerosis (ALS). Eighteen patients with a definite diagnosis of ALS received microinjections of hNSCs into the gray matter tracts of the lumbar or cervical spinal cord. Patients were monitored before and after transplantation by clinical, psychological, neuroradiological, and neurophysiological assessment. For up to 60 months after surgery, none of the patients manifested severe adverse effects or increased disease progression because of the treatment. Eleven patients died, and two underwent tracheotomy as a result of the natural history of the disease. We detected a transitory decrease in progression of ALS Functional Rating Scale Revised, starting within the first month after surgery and up to 4 months after transplantation. Our results show that transplantation of hNSC is a safe procedure that causes no major deleterious effects over the short or long term. This study is the first example of medical transplantation of a highly standardized cell drug product, which can be reproducibly and stably expanded ex vivo, comprising hNSC that are not immortalized, and are derived from the forebrain of the same two donors throughout this entire study as well as across future trials. Our experimental design provides benefits in terms of enhancing both intra- and interstudy reproducibility and homogeneity. Given the potential therapeutic effects of the hNSCs, our observations support undertaking future phase II clinical studies in which increased cell dosages are studied in larger cohorts of patients. Stem Cells Translational Medicine 2019;8:887–897
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There is no specific treatment available for ALS, although novel treatments include gene therapy and stem cell therapy.
“There is no specific treatment available for ALS, although the more popular novel treatments include gene therapy (7) and stem cell therapy (8).”
From Modulation of the gut–brain axis via the gut microbiota: a new era in treatment of amyotrophic lateral sclerosis (2023) · cites this paper as [8]
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A Preview of Selected Articles
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Self-citation
- Authors
- Mazzini, L., Gelati, M., Profico, D. C., 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, M. F., 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, N. M., Vescovi, A. L., & on behalf of the ALS-NSCs Trial Study Group
- Affiliations
- Università degli Studi del Piemonte Orientale “Amedeo Avogadro” (IT)
- Casa Sollievo della Sofferenza (IT)
- d Department of Neuroscience University of Padua, Padua, Italy
- University of Milano-Bicocca (IT)
- Azienda Ospedaliera S.Maria (IT)
- University of Padua (IT)
- Azienda Ospedaliero Universitaria Maggiore della Carita (IT)
- a Eastern Piedmont University, “Maggiore della Carità” Hospital Dipartimento di Neurologia, Novara
- l StemGen SpA, Milan, Italy
- n Fondazione IRCCS Casa Sollievo della Sofferenza Cytogenetics Unit, San Giovanni Rotondo, Foggia, Italy
- Emory University (US)
- Corresponding author
- Mazzini, Letizia
- Vescovi, Angelo L.
- ORCID
- Mazzini, Letizia
- Gelati, Maurizio
- Profico, Daniela Celeste
- Ferrari, Daniela
- Copetti, Massimiliano
- Muzi, Gianmarco
- Ricciolini, Claudia
- Giorgi, Cesare
- Masiero, Stefano
- Stecco, Alessandro
- Cisari, Carlo
- Bersano, Enrica
- Querin, Giorgia
- Cantello, Roberto
- Zalfa, Cristina
- Binda, Elena
- Trombetta, Domenico
- Bernardini, Laura
- Gaiani, Alessandra
- Boulis, Nicholas M.
- Vescovi, Angelo L.
- Venue
- Stem Cells Translational Medicine
- Publisher
- Oxford University Press (OUP)
- Published
- 2019-05-18
- Volume
- 8
- Issue
- 9
- Pages
- 887-897
- ISSN
- 2157-6564 (ISSN-L)
- 2157-6580
- PMC
- PMC6708070
- OpenAlex
- W2945776234
- Cited by
- 128
- References
- 42
- Citations by year
- 2026: 10 · 2025: 21 · 2024: 19 · 2023: 23 · 2022: 14 · 2021: 20 · 2020: 17 · 2019: 3 · 2017: 1
- FWCI
- 9.13
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Stem Cells Translational Medicine (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Padua Research Archive (University of Padova) (submittedVersion, cc-by-nc-nd)
- HAL (submittedVersion)
- BOA (University of Milano-Bicocca) (submittedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Pathology and Forensic Medicine › Spinal Cord Injury Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
- Keywords
- amyotrophic lateral sclerosis, neural stem cells, cell therapy safety, disease progression, ALSFRS-R, long-term outcomes, ex vivo cell expansion
- MeSH terms
- Adult, Aged, Amyotrophic Lateral Sclerosis, Brain, Female, Glial Fibrillary Acidic Protein, Humans, Injections, Spinal, Magnetic Resonance Imaging, Male, Middle Aged, Pain, Pilot Projects, Spinal Cord, Treatment Outcome, Brain-Derived Neurotrophic Factor, Stem Cell Transplantation, Vascular Endothelial Growth Factor A, Young Adult, Neural Stem Cells
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