https://doi.org/10.55788/62267c9c
MSC-NPs are bone marrow-derived cells with trophic and immunomodulatory properties. Their therapeutic potential in MS was evaluated in a randomised, double-blind, placebo-controlled phase 2 trial (NCT03355365) [1]. Participants had either primary progressive disease (PPMS) or secondary progressive disease (SPMS) and a significant disability but could still walk (EDSS 3.0–6.5). The 51 participants were randomised to 6 intrathecal injections of 10 million MSC-NPs (n=24) or saline (n=27) spaced 2 months apart. The compassionate crossover design allowed participants to change to the opposite group in year 2. The primary outcome was EDSS Plus, which means improved EDSS, timed 25-foot walk (T25FW), or 9-hole peg test (9HPT).
After 1 year, no significant differences were seen in the primary endpoint. In the MSC-NP group, EDSS Plus improved in 33% of participants and in the placebo group in 37%. After 2 years, 47 participants received a placebo as well as MSC-NP. In this group, EDSS improved in 20 of 47 participants (43%), 16 participants (34%) remained stable, and 11 (23%) declined.
There were no serious adverse events related to MSC-NP treatment. A total of 7 participants withdrew from the study and no cases of meningitis or malignancies associated with the intervention were observed. Mild headaches and fever were relatively more frequent following MSC-NP treatment.
Regarding secondary endpoints, in participants with EDSS 6.0–6.5, the MSC-NP group performed significantly better than the placebo group on the T25FW (P=0.030), confirmed by the 6-minute walk test (P=0.036). In participants with less advanced grey matter atrophy, grey matter was better preserved in the treatment group (P=0.021). Furthermore, of participants with impaired bladder function, 11 of 16 (69%) in the treatment group had improved post-void residual volume versus 4 of 11 (36%) in the placebo group. A biomarker analysis revealed that MSC-NP was associated with increased matrix metalloproteinase 9 and decreased CC chemokine ligand-2 in the cerebrospinal fluid.
- Sadiq S. Efficacy of intrathecal mesenchymal stem cell-neural progenitor therapy in progressive MS: Results from a phase II clinical trial. S16.005, AAN 2023 Annual Meeting, 22–27 April, Boston, USA.
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Table of Contents: AAN 2023
Featured articles
Letter from the Editor
Positive results for hereditary transthyretin-mediated amyloid polyneuropathy
Infectious Diseases
Allogenic T-cell-based immunotherapy for PML in development
Cognitive Impairment and Dementia
Lecanemab may slow decline of cognition and function in Alzheimer’s Disease
Donanemab shows rapid and deep plaque clearance in early Alzheimer’s Disease
Epilepsy
Seizure forecasting and detection with wearable devices are feasible
Encouraging first results of GABAergic interneurons implants for focal epilepsy
Headache and Migraine
Lecture on migraine: from the prodromal phase to future paradigm shifts
Zavegepant nasal spray exhibits good efficacy and safety in acute migraine
A vaccine as a potentially safe and effective immunotherapy against CGRP
Multiple Sclerosis
Teriflunomide prevents conversion to MS in patients with RIS
Gold nanocrystals may be effective as adjunctive MS therapy
Muscle and Neuro-Muscular Disorders
First-ever ALS platform trial reports on outcomes of 4 treatments
Pridopidine for Huntington’s disease fails to meet the primary endpoint
Parkinson's Disease
Continuous levodopa/carbidopa infusion shows favourable safety and efficacy
Unilateral right STN-DBS improves verbal fluency
Stroke
Harnessing the microbiome as a possible stroke treatment
Patients with a large core infarct benefit from thrombectomy
Miscellaneous
Artificial intelligence applications in neurology: seize the moment
Spinal cord stimulation eases painful diabetic neuropathy
EVT improves functional outcomes in Chinese patients with BAO
Severe sleep apnoea associated with white matter hyperintensities
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