Evidence map›Paper›PMID 40346540›Full record

ArticleJournal of translational medicine2025

Safety and efficacy evaluation of intracerebroventricular human neural stem cell transplantation in SOD1 mice as a novel approach for ALS.

Ivan Lombardi, Clelia Ferrero, Edvige Vulcano, Daniela Maria Rasà, Maurizio Gelati, Diego Pastor, Rose Mary Carletti, Silvia de la Morena, Daniela Celeste Profico, Sabrina Longobardi and 8 more

Abstract read
In one paragraph

Article in Journal of translational medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
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

3 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
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

18 authors.

Ivan LombardiSchool of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy.ORCID 0000-0001-5682-0823
Clelia Ferrero *Neuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy.
Edvige Vulcano *Department of Biotechnology and Biosciences, University of Milano-Bicocca, Milan, Italy.ORCID 0009-0002-4550-3907
Daniela Maria RasàNeuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy.
Maurizio GelatiProduction Unit of Advanced Therapies (UPTA), Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy.
Diego PastorSport Research Centre, Miguel Hernández University, Avinguda de la Universitat d'Elx, Elche, Spain.
Rose Mary CarlettiProduction Unit of Advanced Therapies (UPTA), Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy.
Silvia de la MorenaNeuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy.
Daniela Celeste ProficoProduction Unit of Advanced Therapies (UPTA), Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy.
Sabrina LongobardiDepartment of Biotechnology and Biosciences, University of Milano-Bicocca, Milan, Italy.
Elisa LazzarinoNeuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy.
Elisa PerciballiDepartment of Biotechnology and Biosciences, University of Milano-Bicocca, Milan, Italy.
Jessica Diana RosatiCellular Reprogramming Unit, Fondazione IRCCS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy.
Salvador MartinezInstituto de Neurociencias de Alicante (UMH-CSIC), Universidad Miguel Hernandez, San Juan, Alicante, Spain.
Alessandro VercelliNeuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy.
Angelo Luigi VescoviFaculty of Medicine, Link Campus University, Rome, Italy.
Marina BoidoNeuroscience Institute Cavalieri Ottolenghi (N.I.C.O.), University of Turin, Turin, Italy. marina.boido@unito.it.ORCID 0000-0003-1211-1552
Daniela FerrariDepartment of Biotechnology and Biosciences, University of Milano-Bicocca, Milan, Italy. daniela.ferrari@unimib.it.ORCID 0000-0001-7251-9854

Funding

Fondazione Revert Onlus Fondazione Revert OnlusMinistero della Salute R24-5x1000 to Fondazione IRCCS Casa Sollievo della SofferenzaMinistero dell'Istruzione, dell'Università e della Ricerca Dipartimenti di Eccellenza 2023-2027 to Department of Neuroscience "Rita Levi Montalcini"Università degli Studi di Milano-Bicocca 2019-ATESP-0028
6 · The paper itself

Abstract

backgroundNeural stem cell (NSC) transplantation holds promising therapeutic potential for neurodegenerative disorders like amyotrophic lateral sclerosis (ALS). However, pre-clinical studies and early-phase clinical trials have faced challenges hindering the effective clinical translation of this approach. Crucial hurdles include the side-effects of prolonged immunosuppression, concerns regarding cell origin and transplantation dosage, identification of the most appropriate therapeutic window, and invasiveness of surgical procedures. Here, we assessed the safety and efficacy of intracerebroventricular (ICV) hNSC transplantation as a novel and possibly more effective experimental approach for ALS.

methodsWe evaluated the safety of administering up to 1 × 10

resultsOur study suggests that: (i) a bilateral ICV transplantation of 1 × 10

conclusionsICV transplantation of hNSCs emerges as a safe and promising strategy for ALS, demonstrating potential to delay motor decline and reduce spinal cord microgliosis. However, sustained high-dose immunosuppression is crucial for therapeutic efficacy, emphasizing the need for further optimization to overcome translational challenges and achieve durable clinical benefits.

Indexed as

Amyotrophic Lateral SclerosisNeural Stem CellsStem Cell TransplantationSuperoxide Dismutase-1AnimalsDisease Models, AnimalHumansInjections, IntraventricularMiceMice, TransgenicSpinal CordTreatment OutcomeSuperoxide Dismutase-1ALSCell therapyIntracerebroventricularNeural stem cellsSOD1 modelTransplantation

Identifiers

PMID40346540
PMCPMC12065241

What OpenQuestion holds

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Registered trials

None linked

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.