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

Letizia Mazzini1*, Maurizio Gelati23, Daniela Celeste Profico23, Giada Sgaravizzi2, Massimo Projetti Pensi23, Gianmarco Muzi2, Claudia Ricciolini23, Laura Rota Nodari34, Sandro Carletti5, Cesare Giorgi5, Cristina Spera5, Frondizi Domenico5, Enrica Bersano1, Francesco Petruzzelli3, Carlo Cisari6, Annamaria Maglione3, Maria Francesca Sarnelli1, Alessandro Stecco7, Giorgia Querin8, Stefano Masiero8, Roberto Cantello1, Daniela Ferrari4, Cristina Zalfa4, Elena Binda34, Alberto Visioli4, Domenico Trombetta3, Antonio Novelli3, Barbara Torres3, Laura Bernardini3, Alessandro Carriero7, Paolo Prandi1, Serena Servo1, Annalisa Cerino1, Valentina Cima8, Alessandra Gaiani8, Nicola Nasuelli1, Maurilio Massara6, Jonathan Glass9, Gianni Sorarù8, Nicholas M Boulis10 and Angelo L Vescovi11234*

Journal of Translational Medicine 2015, 13:17 


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.
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.
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.
Trial registration:
Phase I trial, Foetal human neural stem cells, Cell therapy, ALS, Advanced therapies


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