Neural Stem Cell Transplantation in Progressive Multiple Sclerosis: Long-Term Outcomes in Humans
In a long-term follow-up of an observational study involving 11 individuals with progressive multiple sclerosis, no serious adverse events were linked to human fetal neural precursor cell transplantation. Cognition remained stable, although five patients showed new brain lesions. Rates of gray matter atrophy were lower than those seen in patients with progressive disease compared to age-matched controls.
- Tags
- #Neuroprotection
- Most relevant to
- —
- Human efficacy
- Not established from this source
- Mechanism
- Not reported
- Evidence strength
- Limited
- Outcome
- Not reported
Why it matters
These findings suggest that human fetal neural precursor cell transplantation may be safe over the long term and could lead to a stabilization of cognitive function and lower atrophy rates in progressive multiple sclerosis patients.
What this does not prove
The study's retrospective nature limits the strength of its conclusions, making it difficult to draw definitive causative relationships from the associations observed.
Next milestone
No next milestone was established from the available source.
Study facts
- Study design
- Observational
- Participants / samples
- 11 · Number of patients with available long-term follow-up data.
- Randomised
- No
- Controlled
- Not reported
- Primary endpoint met
- Not reported
- Relevant MS type
- PPMS
- Publication date
- 2026-07-22
- Evidence reviewed
- Abstract only
- Regulatory approval
- Not reported
- Research areas
- Other
- Biological target
- neural precursor cells
- Pathway
- Not reported
- Population
- individuals with progressive multiple sclerosis
- Size of effect
- Not reported
- Secondary endpoints
- rates of gray and white matter atrophy; circulating biomarkers of neurodegeneration
- MRI endpoints
- new T2-hyperintense brain lesions
- Biomarkers
- circulating biomarkers of neurodegeneration
- Safety
- No serious adverse events related to cell transplantation occurred
Original sources
- Primary evidenceNeural stem cell transplantation in progressive multiple sclerosis: STEMS study long-term outcomes. ↗DOI: 10.1186/s13287-026-05148-5
Supporting passages (26)
study phasefirst-in-human phase I trial (NCT03269071) investigating neural cell transplantation in progressive multiple sclerosis.
study designMETHODS: 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.
subjectsassessed 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.
speciesassessed 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.
sample sizeLong-term follow-up data were available for 11 of 12 patients.
sample size basisRESULTS: Long-term follow-up data were available for 11 of 12 patients.
randomizedNo serious adverse events related to cell transplantation occurred, confirming long-term safety.
research categoriesThe findings support the long-term safety of fetal neural cell transplantation in progressive multiple sclerosis.
relevant ms typesRates 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.
publication date2026-07-22
interventionhuman fetal neural precursor cell transplantation
comparatorcontrol cohort with progressive disease.
primary endpointBACKGROUND: To evaluate the long-term safety and potential neuroprotective effects of human fetal neural precursor cell transplantation in individuals with progressive multiple sclerosis.
follow upmedian 5.7 years
findingsNo serious adverse events related to cell transplantation occurred, confirming long-term safety.
biological targetsBACKGROUND: To evaluate the long-term safety and potential neuroprotective effects of human fetal neural precursor cell transplantation in individuals with progressive multiple sclerosis.
populationindividuals with progressive multiple sclerosis.
secondary endpointsRates 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.
mri endpointsFive patients developed new T2-hyperintense brain lesions without associated contrast enhancement.
biomarkerscirculating biomarkers of neurodegeneration.
safety signalsNo serious adverse events related to cell transplantation occurred, confirming long-term safety.
limitationsTitle: Neural stem cell transplantation in progressive multiple sclerosis: STEMS study long-term outcomes.
peer reviewedPublication types: Journal Article; Observational Study
subjectsMeSH terms: Humans; Female; Adult; Neural Stem Cells; Male; Middle Aged; Retrospective Studies; Multiple Sclerosis; Stem Cell Transplantation; Multiple Sclerosis, Chronic Progressive; Treatment Outcome; Follow-Up Studies; Atrophy; Magnetic Resonance Imaging
subjectsPublication types: Journal Article; Observational Study
study designPublication types: Journal Article; Observational Study
AI assessment, not yet reviewed by a person · version 2 · Community votes are separate from evidence review.