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Peer-reviewedCC BYStem cell research & therapy

Neural stem cell transplantation in progressive multiple sclerosis: STEMS study long-term outcomes.

  • Maria A Rocca1,2,3
  • Angela Genchi2,4
  • Matteo Azzimonti1,2
  • Irene Gattuso2
  • Elisabetta Pagani1
  • Loredana Storelli1
  • Paolo Vezzulli5
  • Elena Brambilla4
  • Roberto Santangelo2,6
  • Raffaella Greco7
  • Fabio Ciceri3,7
  • Roberto Furlan3,8
4 more authors
  • Lucia Moiola2
  • Andrea Falini3,5
  • Gianvito Martino9,10,11
  • Massimo Filippi12,13,14,15,16
16 affiliations
  1. 1Neuroimaging Research Unit, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  2. 2Neurology Unit, IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20132, Milan, Italy.
  3. 3Vita-Salute San Raffaele University, Milan, Italy.
  4. 4Neuroimmunology Unit, Institute of Experimental Neurology, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  5. 5Neuroradiology Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  6. 6Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  7. 7Haematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  8. 8Clinical Neuroimmunology Unit, Institute of Experimental Neurology, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy.
  9. 9Neurology Unit, IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20132, Milan, Italy. [email protected].
  10. 10Vita-Salute San Raffaele University, Milan, Italy. [email protected].
  11. 11Neuroimmunology Unit, Institute of Experimental Neurology, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy. [email protected].
  12. 12Neuroimaging Research Unit, Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy. [email protected].
  13. 13Neurology Unit, IRCCS San Raffaele Scientific Institute, Via Olgettina, 60, 20132, Milan, Italy. [email protected].
  14. 14Vita-Salute San Raffaele University, Milan, Italy. [email protected].
  15. 15Neurophysiology Service, IRCCS San Raffaele Scientific Institute, Milan, Italy. [email protected].
  16. 16Neurorehabilitation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy. [email protected].
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Abstract

Background

To evaluate the long-term safety and potential neuroprotective effects of human fetal neural precursor cell transplantation in individuals with progressive multiple sclerosis.Methods

This retrospective observational study assessed long-term follow-up data (median 5.7 years) from participants in a first-in-human phase I trial (NCT03269071) investigating neural cell transplantation in progressive multiple sclerosis. Clinical and radiological available data were collected and compared to baseline values and reference populations.Results

Long-term follow-up data were available for 11 of 12 patients. No serious adverse events related to cell transplantation occurred, confirming long-term safety. Although physical disability progressed over time, cognition remained stable compared to baseline. Five patients developed new T2-hyperintense brain lesions without associated contrast enhancement. Long-term gray matter atrophy rates were lower than those observed during the initial two years post-transplantation. Rates of gray and white matter atrophy approximated those of age-matched healthy individuals and patients with early-stage multiple sclerosis, with a trend for a lower atrophy rate compared to a control cohort with progressive disease. These structural findings were consistent with stable levels of circulating biomarkers of neurodegeneration.Conclusions

The findings support the long-term safety of fetal neural cell transplantation in progressive multiple sclerosis. Although exploratory in nature, the stability of cognitive function, reduced brain atrophy, and consistent biomarker profiles support the hypothesis of a possible neuroprotective effect that warrants further investigation in a controlled clinical trial setting.

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Cite

BibTeXRIS
BibTeX
@article{rocca2026neural,
  title = {{Neural stem cell transplantation in progressive multiple sclerosis: STEMS study long-term outcomes.}},
  author = {Maria A Rocca and Angela Genchi and Matteo Azzimonti and Irene Gattuso and Elisabetta Pagani and Loredana Storelli and Paolo Vezzulli and Elena Brambilla and Roberto Santangelo and Raffaella Greco and Fabio Ciceri and Roberto Furlan and Lucia Moiola and Andrea Falini and Gianvito Martino and Massimo Filippi},
  journal = {Stem cell research \& therapy},
  year = {2026},
  month = jul,
  doi = {10.1186/s13287-026-05148-5},
  url = {https://europepmc.org/article/MED/42897668},
  note = {CC BY},
}
RIS
TY  - JOUR
TI  - Neural stem cell transplantation in progressive multiple sclerosis: STEMS study long-term outcomes.
AU  - Maria A Rocca
AU  - Angela Genchi
AU  - Matteo Azzimonti
AU  - Irene Gattuso
AU  - Elisabetta Pagani
AU  - Loredana Storelli
AU  - Paolo Vezzulli
AU  - Elena Brambilla
AU  - Roberto Santangelo
AU  - Raffaella Greco
AU  - Fabio Ciceri
AU  - Roberto Furlan
AU  - Lucia Moiola
AU  - Andrea Falini
AU  - Gianvito Martino
AU  - Massimo Filippi
PY  - 2026
DA  - 2026/07/01
JO  - Stem cell research & therapy
DO  - 10.1186/s13287-026-05148-5
UR  - https://europepmc.org/article/MED/42897668
AB  - Background  To evaluate the long-term safety and potential neuroprotective effects of human fetal neural precursor cell transplantation in individuals with progressive multiple sclerosis.Methods  This retrospective observational study assessed long-term follow-up data (median 5.7 years) from participants in a first-in-human phase I trial (NCT03269071) investigating neural cell transplantation in progressive multiple sclerosis. Clinical and radiological available data were collected and compared to baseline values and reference populations.Results  Long-term follow-up data were available for 11 of 12 patients. No serious adverse events related to cell transplantation occurred, confirming long-term safety. Although physical disability progressed over time, cognition remained stable compared to baseline. Five patients developed new T2-hyperintense brain lesions without associated contrast enhancement. Long-term gray matter atrophy rates were lower than those observed during the initial two years post-transplantation. Rates of gray and white matter atrophy approximated those of age-matched healthy individuals and patients with early-stage multiple sclerosis, with a trend for a lower atrophy rate compared to a control cohort with progressive disease. These structural findings were consistent with stable levels of circulating biomarkers of neurodegeneration.Conclusions  The findings support the long-term safety of fetal neural cell transplantation in progressive multiple sclerosis. Although exploratory in nature, the stability of cognitive function, reduced brain atrophy, and consistent biomarker profiles support the hypothesis of a possible neuroprotective effect that warrants further investigation in a controlled clinical trial setting.
SN  - 1757-6512
ER  -