Intrathecal Mesenchymal Stem Cells in Progressive Multiple Sclerosis: A Randomized, Double-Blind, Placebo-Controlled...

Area of Science:

• Neuroscience
• Immunology
• Regenerative Medicine

Background:

• Multiple Sclerosis (MS) presents an unmet need for neuroregenerative therapies.
• Mesenchymal stem cells (MSCs) demonstrate immunomodulatory and regenerative potential in preclinical and early clinical studies.
• Intrathecal administration of MSCs may enhance therapeutic delivery to the central nervous system (CNS).

Purpose of the Study:

• To evaluate the neuroregenerative potential of a single intrathecal administration of autologous MSCs in progressive MS.
• To assess the safety and efficacy of this therapeutic approach through a randomized, placebo-controlled trial.

Main Methods:

• A randomized, double-blind, placebo-controlled phase I/II trial involving patients with progressive MS.
• Single intrathecal injection of autologous bone marrow-derived MSCs.
• Primary endpoint: change in combined evoked potentials latency at 6 months; secondary endpoints: safety, MRI, functional, ophthalmologic, and biomarker assessments.

Main Results:

• No significant neuroregenerative effect was observed based on the primary endpoint.
• A trend towards reduced cerebral atrophy and lower serum glial fibrillary acidic protein levels at 6 months, not sustained at 12 months.
• Adverse events included fever, low back pain, spinal MRI abnormalities, and one case of arachnoiditis, suggesting localized inflammation.

Conclusions:

• A single intrathecal administration of autologous MSCs did not demonstrate a neuroregenerative effect in progressive MS.
• The observed adverse events indicate a potential for acute localized inflammatory reactions.
• Future studies involving intrathecal MSC administration for progressive MS should proceed with caution.