Evidence map›Paper›PMID 31104357›Full record

ArticleStem cells translational medicine2019

Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long-Term Outcome.

Letizia Mazzini, Maurizio Gelati, Daniela Celeste Profico, Gianni Sorarù, Daniela Ferrari, Massimiliano Copetti, Gianmarco Muzi, Claudia Ricciolini, Sandro Carletti, Cesare Giorgi and 24 more

Open access · goldAbstract readClinical Trial, Phase I
In one paragraph

Article in Stem cells translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 68 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
68citing papers in PubMed, 2 pooled it
8.6field-weighted citation impact, top 2% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

68 citing papers in PubMed, 2 syntheses or guidelines pooled it, 124 citations in OpenAlex.

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8 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

34 authors at 9 institutions in 3 countries.

Letizia MazziniEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Maurizio GelatiLaboratorio Cellule Staminali, Cell Factory e Biobanca, Terni Hospital, Italy.
Daniela Celeste ProficoFondazione IRCCS Casa Sollievo della Sofferenza, Advanced Therapies Production Unit, San Giovanni Rotondo, Foggia, Italy.ORCID 0000-0001-5721-0410
Gianni SorarùDepartment of Neuroscience, University of Padua, Padua, Italy.
Daniela FerrariBiotechnology and Bioscience Department Bicocca University, Milan, Italy.
Massimiliano CopettiFondazione IRCCS Casa Sollievo della Sofferenza, Biostatistic Unit, San Giovanni Rotondo, Foggia, Italy.
Gianmarco MuziLaboratorio Cellule Staminali, Cell Factory e Biobanca, Terni Hospital, Italy.
Claudia RiccioliniLaboratorio Cellule Staminali, Cell Factory e Biobanca, Terni Hospital, Italy.
Sandro CarlettiDepartment of Neurosurgery and Neuroscience, "Santa Maria" Hospital, Terni, Italy.
Cesare GiorgiDepartment of Neurosurgery and Neuroscience, "Santa Maria" Hospital, Terni, Italy.
Cristina SperaDepartment of Neurosurgery and Neuroscience, "Santa Maria" Hospital, Terni, Italy.
Domenico FrondiziDepartment of Neurosurgery and Neuroscience, "Santa Maria" Hospital, Terni, Italy.
Stefano MasieroDepartment of Neuroscience, University of Padua, Padua, Italy.
Alessandro SteccoDepartment of Diagnostic and Interventional Radiology, "Eastern Piedmont" University, "Maggiore della Carità" Hospital, Novara.
Carlo CisariDepartment of Physical Therapy, "Eastern Piedmont" University, "Maggiore della Carità" Hospital, Novara.
Enrica BersanoEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Fabiola De MarchiEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Maria Francesca SarnelliEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Giorgia QuerinDepartment of Neuroscience, University of Padua, Padua, Italy.
Roberto CantelloEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Francesco PetruzzelliFondazione IRCCS Casa Sollievo della Sofferenza, Obstetrics and Gynaecology Department, San Giovanni Rotondo, Foggia, Italy.
Annamaria MaglioneFondazione IRCCS Casa Sollievo della Sofferenza, Obstetrics and Gynaecology Department, San Giovanni Rotondo, Foggia, Italy.
Cristina ZalfaBiotechnology and Bioscience Department Bicocca University, Milan, Italy.
Elena BindaFondazione IRCCS Casa Sollievo della Sofferenza, Cancer Stem Cells Unit, San Giovanni Rotondo, Foggia, Italy.
Alberto VisioliStemGen SpA, Milan, Italy.
Domenico TrombettaFondazione IRCCS Casa Sollievo della Sofferenza, Department of Oncology, San Giovanni Rotondo, Foggia, Italy.
Barbara TorresFondazione IRCCS Casa Sollievo della Sofferenza, Cytogenetics Unit, San Giovanni Rotondo, Foggia, Italy.
Laura BernardiniFondazione IRCCS Casa Sollievo della Sofferenza, Cytogenetics Unit, San Giovanni Rotondo, Foggia, Italy.
Alessandra GaianiDepartment of Neuroscience, University of Padua, Padua, Italy.
Maurilio MassaraEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Silvia PaolucciEastern Piedmont University, "Maggiore della Carità" Hospital, Dipartimento di Neurologia, Novara.
Nicholas M BoulisDepartment of Neurosurgery, Emory University, Atlanta, USA.
Angelo L VescoviLaboratorio Cellule Staminali, Cell Factory e Biobanca, Terni Hospital, Italy.
ALS-NSCs Trial Study Group
Piedmont University · USCasa Sollievo della Sofferenza · ITSanta Maria Nuova Hospital · ITUniversity of Padua · ITCell Medica (Switzerland) · CHIstituti di Ricovero e Cura a Carattere Scientifico · ITUniversity of Milano-Bicocca · ITBiogen (Italy) · ITEmory University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

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.

Indexed as

AdultAgedAmyotrophic Lateral SclerosisBrainBrain-Derived Neurotrophic FactorFemaleGlial Fibrillary Acidic ProteinHumansInjections, SpinalMagnetic Resonance ImagingMaleMiddle AgedNeural Stem CellsPainPilot ProjectsSpinal CordBDNF protein, humanBrain-Derived Neurotrophic FactorGlial Fibrillary Acidic ProteinVascular Endothelial Growth Factor AAdult stem cellsCellular therapyClinical trialsFetal stem cells

Identifiers

PMID31104357
PMCPMC6708070
OpenAlexW2945776234

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.