What the study found
A single intrathecal administration of autologous mesenchymal stem cells (MSCs, cells given into the spinal fluid) did not show a neuroregenerative effect in progressive multiple sclerosis. The authors classify this as Class III evidence.
Why the authors say this matters
The study suggests there is still an unmet need for neuroregenerative therapies in multiple sclerosis, and the authors conclude that intrathecal MSC treatment in progressive MS should be approached with caution in future studies.
What the researchers tested
This randomized, placebo-controlled trial tested whether one intrathecal dose of autologous MSCs in people with progressive MS could produce evidence of neuroregeneration. The trial used a crossover design and measured a primary endpoint at 6 months, with secondary outcomes including safety, brain MRI, functional and ophthalmologic assessments, and serum biomarkers at 6 and 12 months; exploratory CSF proteomics were also analyzed.
What worked and what didn't
The primary composite evoked potential outcome showed no neuroregenerative effect. Some MRI and functional measures improved at 6 months, but these changes were not sustained at 12 months, and exploratory CSF proteomics showed reductions in multiple inflammation-related proteins at 6 months.
What to keep in mind
The abstract notes that interpretation may be limited by the small sample size. One serious adverse event was probably related to treatment, and other adverse events included fever, low back pain, spinal MRI abnormalities with fluid loculations and nerve root clumping, and one case of chronic coccygeal pain attributed to arachnoiditis.
Key points
- A single intrathecal dose of autologous MSCs did not show a neuroregenerative effect in progressive multiple sclerosis.
- The primary endpoint was change in latency of combined evoked potentials at 6 months.
- Some MRI and functional findings improved at 6 months but were not sustained at 12 months.
- Exploratory CSF proteomics showed reductions in multiple inflammation-related proteins at 6 months.
- Adverse events included fever, low back pain, spinal MRI abnormalities, and one probable treatment-related serious adverse event.
Disclosure
- Research title:
- Intrathecal MSCs did not show neuroregenerative effect in progressive MS
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