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Author Notes:

Correspondence: mazzini.l@libero.it; vescovi@tin.it

Letizia Mazzini and Maurizio Gelati contributed equally to this work.

See publication for full list of author contributions.

We thank all the patients participating in the trial and their families, the doctors, nurses, physical therapists at “Santa Maria”, “Maggiore della Carità” and “San Antonio” Hospitals and Stefania Moia for the organizative support.

The authors declare that they have no competing interests.


Research Funding:

This trial was supported by the Revert Onlus Association and the Fondazione Cellule Staminali di Terni.

The Fondazione Stefano Borgonovo also supported our research.

Special thanks go to the "ASSICURAZIONI GENERALI" group, whose continued sponsorship was fundamental to the success of this research.


  • Science & Technology
  • Life Sciences & Biomedicine
  • Medicine, Research & Experimental
  • Research & Experimental Medicine
  • Phase I trial
  • Foetal human neural stem cells
  • Cell therapy
  • ALS
  • Advanced therapies

Human neural stem cell transplantation in ALS: initial results from a phase I trial

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Journal Title:

Journal of Translational Medicine


Volume 13, Number 1


, Pages 17-17

Type of Work:

Article | Final Publisher PDF


Background: We report the initial results from a phase I clinical trial for ALS. We transplanted GMP-grade, fetal human neural stem cells from natural in utero death (hNSCs) into the anterior horns of the spinal cord to test for the safety of both cells and neurosurgical procedures in these patients. The trial was approved by the Istituto Superiore di Sanità and the competent Ethics Committees and was monitored by an external Safety Board. Methods: Six non-ambulatory patients were treated. Three of them received 3 unilateral hNSCs microinjections into the lumbar cord tract, while the remaining ones received bilateral (n = 3 + 3) microinjections. None manifested severe adverse events related to the treatment, even though nearly 5 times more cells were injected in the patients receiving bilateral implants and a much milder immune-suppression regimen was used as compared to previous trials. Results: No increase of disease progression due to the treatment was observed for up to18 months after surgery. Rather, two patients showed a transitory improvement of the subscore ambulation on the ALS-FRS-R scale (from 1 to 2). A third patient showed improvement of the MRC score for tibialis anterior, which persisted for as long as 7 months. The latter and two additional patients refused PEG and invasive ventilation and died 8 months after surgery due to the progression of respiratory failure. The autopsies confirmed that this was related to the evolution of the disease. Conclusions: We describe a safe cell therapy approach that will allow for the treatment of larger pools of patients for later-phase ALS clinical trials, while warranting good reproducibility. These can now be carried out under more standardized conditions, based on a more homogenous repertoire of clinical grade hNSCs. The use of brain tissue from natural miscarriages eliminates the ethical concerns that may arise from the use of fetal material.

Copyright information:

© 2015 Mazzini et al.

This is an Open Access work distributed under the terms of the Creative Commons Attribution 4.0 International License (https://creativecommons.org/licenses/by/4.0/).
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