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Article Dans Une Revue Stem Cells Translational Medicine Année : 2019

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

Maurizio Gelati
  • Fonction : Auteur
Daniela Celeste Profico
  • Fonction : Auteur
Daniela Ferrari
  • Fonction : Auteur
Gianmarco Muzi
  • Fonction : Auteur
Claudia Ricciolini
  • Fonction : Auteur
Sandro Carletti
  • Fonction : Auteur
Cesare Giorgi
  • Fonction : Auteur
Cristina Spera
  • Fonction : Auteur
Domenico Frondizi
  • Fonction : Auteur
Carlo Cisari
  • Fonction : Auteur
Enrica Bersano
  • Fonction : Auteur
Maria Francesca Sarnelli
  • Fonction : Auteur
Roberto Cantello
  • Fonction : Auteur
Francesco Petruzzelli
  • Fonction : Auteur
Annamaria Maglione
  • Fonction : Auteur
Cristina Zalfa
  • Fonction : Auteur
Elena Binda
  • Fonction : Auteur
Alberto Visioli
  • Fonction : Auteur
Barbara Torres
  • Fonction : Auteur
Laura Bernardini
  • Fonction : Auteur
Alessandra Gaiani
  • Fonction : Auteur
Maurilio Massara
  • Fonction : Auteur
Silvia Paolucci
  • Fonction : Auteur
Nicholas Boulis
  • Fonction : Auteur
Angelo Vescovi
  • Fonction : Auteur

Résumé

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

Dates et versions

hal-03971730 , version 1 (03-02-2023)

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Citer

Letizia Mazzini, Maurizio Gelati, Daniela Celeste Profico, Gianni Sorarù, Daniela Ferrari, et al.. Results from Phase I Clinical Trial with Intraspinal Injection of Neural Stem Cells in Amyotrophic Lateral Sclerosis: A Long-Term Outcome. Stem Cells Translational Medicine, 2019, 8 (9), pp.887-897. ⟨10.1002/sctm.18-0154⟩. ⟨hal-03971730⟩
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